256. 筋型糖原病 Disease details / Clinical trials / Drug dev / DR info

1-deoxynojirimycin hydrochloride
   Amicus Therapeutics, Inc.
      2011   -   EUCTR2011-002154-32-GB   Canada;France;United Kingdom;United States
      2009   -   EUCTR2008-002302-18-DE   Germany;United Kingdom
      2008   -   EUCTR2008-002302-18-GB   Germany;United Kingdom
ABX1100 injection FOR IV infusion
   Aro Biotherapeutics
      2023   Early Phase 1   NCT06109948   Canada;United States
Acetaminophen
   University of Florida
      2017   Phase 1   NCT02240407   United States
Actus-101
   Asklepios Biopharmaceutical, Inc.
      2018   Phase 1/Phase 2   NCT03533673   United States
Albuterol
   Duke University
      2013   Phase 1/Phase 2   NCT01885936   United States
      2012   Phase 1   NCT01859624   United States
Aldurazyme
   University of California, San Francisco
      2021   Phase 1   NCT04532047   United States
Alglucosidase alfa
      -   Phase 3   EUCTR2019-002251-42-NL   Netherlands
   Amicus Therapeutics
      2018   Phase 3   NCT03729362   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Poland;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
   Amicus Therapeutics, Inc.
      2019   Phase 3   EUCTR2018-000755-40-SK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-SI   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-SE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-NL   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-HU   Australia;Austria;Belgium;Bosnia and Herzegovina;Brazil;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-GR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-GB   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-ES   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-DK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-DE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-BG   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-BE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-AT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2018   Phase 4   EUCTR2017-004370-34-HU   Australia;Belgium;Brazil;Bulgaria;Canada;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Democratic People's Republic of;Netherlands;Poland;Romania;Slovakia;Spain;Sweden;Taiwan;United Kingdom;United States
      2018   Phase 4   EUCTR2017-004370-34-BE   Australia;Belgium;Brazil;Canada;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Democratic People's Republic of;Netherlands;Spain;Sweden;Taiwan;United Kingdom;United States
      -   Phase 2   EUCTR2013-002257-30-GB   Netherlands;United Kingdom;United States
   Amicus Therapeutics, Inc./CMIC Co., Ltd.
      2019   Phase 3   JPRN-jRCT2080224804   Asia except Japan;Europe;Japan;North America;Oceania;South America
   Erasmus MC Sophia
      2009   -   EUCTR2008-008293-31-NL   Netherlands
   GENZYME
      2004   -   EUCTR2004-002168-59-IT   Italy
   GENZYME CORPORATION
      2016   Phase 3   EUCTR2016-000942-77-IT   Austria;Belgium;Czech Republic;Denmark;Germany;Italy;Netherlands;Poland;Spain;Sweden;United Kingdom
   Genzyme
      -   Phase 4   EUCTR2024-000461-24-Outside-EU/EEA   China
   Genzyme Corporation
      2019   Phase 3   EUCTR2016-000942-77-HU   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2016-000942-77-PT   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2016-000942-77-BG   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2017   Phase 2   EUCTR2016-003475-21-GB   France;Germany;Japan;Taiwan;United Kingdom;United States
      2017   Phase 3   EUCTR2016-000942-77-NL   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2016-000942-77-AT   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Türkiye;United Kingdom;United States
      2017   Phase 4   EUCTR2010-022231-11-GB   Bulgaria;Germany;India;Russian Federation;Ukraine;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-SE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-GB   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-ES   Argentina;Austria;Belgium;Brazil;Canada;Colombia;Czech Republic;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-DK   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-DE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-CZ   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-BE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2012   Phase 1;Phase 4   EUCTR2010-022231-11-DE   Bulgaria;China;Germany;India;Poland;Russian Federation;Ukraine;United Kingdom;United States
      -   Phase 4   EUCTR2021-005552-11-Outside-EU/EEA   United States
      -   Phase 2   EUCTR2016-003475-21-FR   France;Germany;Japan;Taiwan;United Kingdom;United States
      -   Phase 2   EUCTR2016-003475-21-DE   France;Germany;Japan;Taiwan;United Kingdom;United States
      -   Phase 3   EUCTR2016-000942-77-PL   Argentina;Australia;Austria;Belgium;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
   Genzyme Corporation Inc
      -   -   EUCTR2015-000584-14-Outside-EU/EEA   United States
      -   -   EUCTR2015-000583-34-Outside-EU/EEA   Israel;United States
      -   -   EUCTR2015-000582-31-Outside-EU/EEA   Australia;Canada;United States
      -   -   EUCTR2015-000512-18-Outside-EU/EEA   United States
   Genzyme Corporation, a Sanofi company
      2013   Phase 4   EUCTR2011-005595-42-DE   Canada;Germany;India;Russian Federation;Taiwan;Turkey;United States
   Genzyme Europe B.V.
      2012   -   EUCTR2010-020611-36-NL   Denmark;Germany;Netherlands;United Kingdom;United States
      2011   -   EUCTR2010-020611-36-GB   Germany;Netherlands;United Kingdom;United States
      2011   -   EUCTR2010-020611-36-DE   Germany;Netherlands;United Kingdom;United States
      2007   -   EUCTR2006-003644-31-DE   France;Germany;Netherlands
   Genzyme Europe BV
      2007   -   EUCTR2006-003644-31-NL   France;Germany;Netherlands
      2007   Phase 4   EUCTR2006-003644-31-FR   France;Germany;Netherlands
      2007   -   EUCTR2005-002759-42-DE   Germany
      2005   Phase 2   EUCTR2005-002829-31-GB   Belgium;United Kingdom
      2005   -   EUCTR2005-001629-27-DE   Germany;Italy
      -   Phase 2   EUCTR2005-002829-31-BE   Belgium;United Kingdom
   Genzyme, a Sanofi Company
      2021   Phase 4   NCT04676373   China
      2018   Phase 4   NCT03687333   China
      2017   Phase 2   NCT03019406   France;Japan;Taiwan;United Kingdom;United States
      2016   Phase 3   NCT02782741   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2015   -   NCT01710813   Belgium;Czech Republic;Czechia;Germany;Italy;Taiwan;United States
      2014   Phase 4   NCT01410890   Bulgaria;Germany;India;Russian Federation;Ukraine;United Kingdom;United States
      2012   Phase 4   NCT01597596   Germany;Taiwan;United States
      2012   Phase 4   NCT01526785   United States
      2012   -   NCT00566878   United States
      2011   Phase 4   NCT01288027   Germany;Netherlands;United Kingdom;United States
      2009   Phase 4   NCT00701129   Israel;United States
      2008   Phase 4   NCT00701701   Canada;Israel;United States
      2008   Phase 4   NCT00486889   United States
      2007   -   NCT00520143   United States
      2007   Phase 4   NCT00483379   Australia;Canada;United States
      2007   Phase 4   NCT00455195   Australia;Canada;France;Netherlands;United States
      2005   Phase 3   NCT00158600   France;Netherlands;United States
      2003   -   NCT00074919   United States
      2003   Phase 2   NCT00051935   United States
   Sanofi(China) Investment Co Ltd, Shanghai Branch
      -   Phase 4   EUCTR2021-004047-25-Outside-EU/EEA   China
   Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of Medicine
      2018   Phase 4 study   ChiCTR1800018514   China
   Taipei Veterans General Hospital, Taiwan
      2021   -   NCT05017402   -
   Valerion Therapeutics, LLC
      2017   Phase 1;Phase 2   EUCTR2016-004578-16-GB   United Kingdom;United States
Alglucosidase alfa GZ419829
   Sanofi
      2021   -   NCT04848779   Belgium;France;Germany;Italy;Netherlands;Spain;Taiwan;United Kingdom;United States
Alglucosidase alfa or avalglucosidase alfa
   Amicus Therapeutics
      2024   -   NCT06121011   Belgium;Germany;United Kingdom;United States
Alglucosidase alpha
   Genzyme Europe B.V.
      2007   -   EUCTR2006-003644-31-DE   France;Germany;Netherlands
   Genzyme Europe BV
      2007   -   EUCTR2006-003644-31-NL   France;Germany;Netherlands
      2007   Phase 4   EUCTR2006-003644-31-FR   France;Germany;Netherlands
      2007   -   EUCTR2005-002759-42-DE   Germany
      2005   Phase 2   EUCTR2005-002829-31-GB   Belgium;United Kingdom
      -   Phase 2   EUCTR2005-002829-31-BE   Belgium;United Kingdom
Alglucosidasi alfa umana ricombinante
   AMICUS THERAPEUTICS, INC.
      2019   Phase 3   EUCTR2018-000755-40-IT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Democratic People's Republic of;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
Algucosidase alfa 20 MG/KG once every 4 weeks instead OF once every 2 weeks
   Erasmus Medical Center
      2024   Phase 4   NCT06575829   -
Alpha-glucosidase
   Genzyme, a Sanofi Company
      2007   -   NCT00520143   United States
      2001   Phase 2   NCT00025896   United States
AT2220
   Amicus Therapeutics, Inc.
      2011   -   EUCTR2011-002154-32-GB   Canada;France;United Kingdom;United States
      2009   -   EUCTR2008-002302-18-DE   Germany;United Kingdom
      2008   -   EUCTR2008-002302-18-GB   Germany;United Kingdom
      -   Phase 2   EUCTR2013-002257-30-GB   Netherlands;United Kingdom;United States
AT2221
   AMICUS THERAPEUTICS, INC.
      2020   Phase 3   EUCTR2019-000954-67-IT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Democratic People's Republic of;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-IT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Democratic People's Republic of;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
   Amicus Therapeutics
      2020   -   NCT04327973   Italy;Taiwan;United States
      2019   Phase 3   NCT04138277   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Poland;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   -   NCT03865836   -
      2018   Phase 3   NCT03729362   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Poland;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2016   Phase 1/Phase 2   NCT02675465   Australia;Germany;Netherlands;New Zealand;United Kingdom;United States
   Amicus Therapeutics, Inc.
      2020   Phase 3   EUCTR2019-000954-67-SI   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-GR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-GB   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-FR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-ES   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-DK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-DE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-BE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-SK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-SI   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-SE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-NL   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-HU   Australia;Austria;Belgium;Bosnia and Herzegovina;Brazil;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-GR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-GB   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-ES   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-DK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-DE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-BG   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-BE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-AT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2016   Phase 1;Phase 2   EUCTR2015-004798-34-NL   Australia;Germany;Netherlands;New Zealand;United Kingdom;United States
      2016   Phase 1;Phase 2   EUCTR2015-004798-34-GB   Australia;Germany;Netherlands;United Kingdom;United States
      2016   Phase 1;Phase 2   EUCTR2015-004798-34-DE   Australia;Germany;Netherlands;New Zealand;United Kingdom;United States
   Amicus Therapeutics, Inc./CMIC Co., Ltd.
      2019   Phase 3   JPRN-jRCT2080224804   Asia except Japan;Europe;Japan;North America;Oceania;South America
   Wright Jacquelyn
      2021   -   JPRN-jRCT2061210056   -
      2021   Phase 3   JPRN-jRCT2031210014   Canada;Japan;Taiwan;United States of America
      2020   Phase 3   JPRN-jRCT2011200003   Asia;Europe;Japan;North America;Oceania;South America
AT2221 65 MG formulated capsules
   Amicus Therapeutics, Inc.
      2020   Phase 3   EUCTR2019-000954-67-AT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
AT845
   Audentes Therapeutics, Inc.
      2020   Phase 1;Phase 2   EUCTR2019-003595-38-GB   Germany;Italy;United Kingdom
ATB200
   AMICUS THERAPEUTICS, INC.
      2020   Phase 3   EUCTR2019-000954-67-IT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Democratic People's Republic of;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-IT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Democratic People's Republic of;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
   Amicus Therapeutics
      2020   -   NCT04327973   Italy;Taiwan;United States
      2019   Phase 3   NCT04138277   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Poland;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   -   NCT03865836   -
      2018   Phase 3   NCT03729362   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Poland;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2016   Phase 1/Phase 2   NCT02675465   Australia;Germany;Netherlands;New Zealand;United Kingdom;United States
   Amicus Therapeutics, Inc.
      2020   Phase 3   EUCTR2019-000954-67-SI   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-GR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-GB   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-FR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-ES   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-DK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-DE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-BE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-AT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-SK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-SI   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-SE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-NL   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-HU   Australia;Austria;Belgium;Bosnia and Herzegovina;Brazil;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-GR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-GB   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-ES   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-DK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-DE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-BG   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-BE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-AT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2016   Phase 1;Phase 2   EUCTR2015-004798-34-NL   Australia;Germany;Netherlands;New Zealand;United Kingdom;United States
      2016   Phase 1;Phase 2   EUCTR2015-004798-34-GB   Australia;Germany;Netherlands;United Kingdom;United States
      2016   Phase 1;Phase 2   EUCTR2015-004798-34-DE   Australia;Germany;Netherlands;New Zealand;United Kingdom;United States
   Amicus Therapeutics, Inc./CMIC Co., Ltd.
      2019   Phase 3   JPRN-jRCT2080224804   Asia except Japan;Europe;Japan;North America;Oceania;South America
   Wright Jacquelyn
      2021   -   JPRN-jRCT2061210056   -
      2021   Phase 3   JPRN-jRCT2031210014   Canada;Japan;Taiwan;United States of America
      2020   Phase 3   JPRN-jRCT2011200003   Asia;Europe;Japan;North America;Oceania;South America
Avalglucosidase alfa
      -   Phase 3   EUCTR2019-002251-42-NL   Netherlands
   Genzyme Corporation
      2017   Phase 2   EUCTR2016-003475-21-GB   France;Germany;Japan;Taiwan;United Kingdom;United States
      2014   Phase 2   EUCTR2013-003321-28-DK   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States
      2014   Phase 2   EUCTR2013-003321-28-DE   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States
      2014   Phase 2   EUCTR2013-003321-28-BE   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States
   Genzyme, a Sanofi Company
      2025   Phase 4   NCT06666413   -
      2022   Phase 4   NCT05164055   France
      2017   Phase 2   NCT03019406   France;Japan;Taiwan;United Kingdom;United States
      2016   Phase 3   NCT02782741   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2014   Phase 2   NCT02032524   Belgium;Denmark;France;Germany;Netherlands;United Kingdom;United States
   SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
      2021   Phase 3   EUCTR2020-004686-39-IT   Belgium;China;France;Germany;Italy;Netherlands;Spain;Taiwan;United Kingdom;United States
   Sanofi
      2021   Phase 3   NCT04910776   Belgium;China;Germany;Italy;Netherlands;Spain;Taiwan;United Kingdom;United States
   Sanofi-Aventis Recherche & Développement
      2021   Phase 3   EUCTR2020-004686-39-NL   Belgium;China;France;Germany;Italy;Netherlands;Spain;Taiwan;United Kingdom;United States
      2021   Phase 3   EUCTR2020-004686-39-BE   Belgium;China;France;Germany;Italy;Netherlands;Spain;Taiwan;United Kingdom;United States
      -   Phase 4   EUCTR2021-002590-26-FR   France
      -   Phase 3   EUCTR2020-004686-39-DE   Belgium;China;France;Germany;Italy;Netherlands;Spain;Taiwan;United Kingdom;United States
Avalglucosidase ALFA-ngpt (GZ402666) IV
   Sanofi
      2022   -   NCT05734521   United States
Avalglucosidasi alfa
   SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
      2021   Phase 3   EUCTR2020-004686-39-IT   Belgium;China;France;Germany;Italy;Netherlands;Spain;Taiwan;United Kingdom;United States
Blood draw FOR optional genetic exome sequencing
   LMU Klinikum
      2022   -   NCT05272969   Germany
BMN 701
   BioMarin Pharmaceutical
      2014   Phase 3   NCT01924845   Belgium;France;Germany;Italy;Netherlands;Portugal;United Kingdom;United States
      2011   Phase 2   NCT01435772   Australia;France;Germany;New Zealand;United Kingdom;United States
      2011   Phase 1/Phase 2   NCT01230801   Australia;France;Germany;United Kingdom;United States
   BioMarin Pharmaceutical Inc
      2016   Phase 3   EUCTR2013-001768-48-AT   Austria;Belgium;France;Germany;Italy;Japan;Netherlands;Portugal;Switzerland;United Kingdom;United States
      2015   Phase 3   EUCTR2013-001768-48-PT   Argentina;Austria;Belgium;Colombia;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States
      2014   Phase 3   EUCTR2013-001768-48-IT   Argentina;Austria;Belgium;Colombia;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States
      2014   Phase 3   EUCTR2013-001768-48-DE   Argentina;Austria;Belgium;Colombia;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States
      2014   Phase 3   EUCTR2013-001768-48-BE   Argentina;Austria;Belgium;Colombia;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States
      2013   Phase 3   EUCTR2013-001768-48-GB   Austria;Belgium;Colombia;Denmark;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States
      2013   Phase 2   EUCTR2011-001805-28-DE   Australia;Germany;New Zealand;United Kingdom;United States
      2012   Phase 2   EUCTR2011-001805-28-GB   Australia;France;Germany;United Kingdom;United States
      2012   -   EUCTR2010-023561-22-DE   France;Germany;Netherlands;United Kingdom;United States
      2011   Phase 1;Phase 2   EUCTR2010-023561-22-GB   Germany;United Kingdom;United States
Borg scale
   LMU Klinikum
      2022   -   NCT05272969   Germany
Bortezomib
   National Taiwan University Hospital
      2015   Phase 4   NCT02525172   Taiwan
Cipaglucosidase alfa
   Amicus Therapeutics
      2024   -   NCT06121011   Belgium;Germany;United Kingdom;United States
      2023   Phase 3   NCT04808505   France;Germany;Italy;Netherlands;Taiwan;United Kingdom;United States
      2020   Phase 3   NCT03911505   Australia;Canada;Germany;Italy;Japan;Taiwan;United States
   Amicus Therapeutics, Inc.
      2020   Phase 3   EUCTR2019-000954-67-SI   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-GR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-GB   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-FR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-DK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-DE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-BE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-AT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-SI   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-SE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-NL   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-GR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-DK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-DE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-BE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-AT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2016   Phase 1;Phase 2   EUCTR2015-004798-34-GB   Australia;Germany;Netherlands;United Kingdom;United States
      2016   Phase 1;Phase 2   EUCTR2015-004798-34-DE   Australia;Germany;Netherlands;New Zealand;United Kingdom;United States
Cipaglucosidasi alfa
   AMICUS THERAPEUTICS, INC.
      2020   Phase 3   EUCTR2019-000954-67-IT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Democratic People's Republic of;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
Clenbuterol
   Dwight Koeberl, M.D., Ph.D.
      2013   Phase 1/Phase 2   NCT01942590   United States
CRG003 injection
   Huashan Hospital
      2023   Early Phase 1   NCT06178432   China
Cyclophosphamide monohydrate
   Genzyme Corporation Inc
      -   -   EUCTR2015-000583-34-Outside-EU/EEA   Israel;United States
Deprakine
   John Vissing
      2014   Phase 2   EUCTR2014-001637-88-DK   Denmark
Diphenhydramine
   University of Florida
      2017   Phase 1   NCT02240407   United States
Duvoglustat
   Amicus Therapeutics
      2011   Phase 2   NCT01380743   Canada;France;United Kingdom;United States
      2008   Phase 2   NCT00688597   Australia;Canada;France;Germany;Netherlands;United Kingdom;United States
Duvoglustat HCL
   Amicus Therapeutics, Inc.
      -   Phase 2   EUCTR2013-002257-30-GB   Netherlands;United Kingdom;United States
Duvoglustat hydrochloride
   Amicus Therapeutics, Inc.
      2011   -   EUCTR2011-002154-32-GB   Canada;France;United Kingdom;United States
      -   Phase 2   EUCTR2013-002257-30-GB   Netherlands;United Kingdom;United States
Enzyme replacement agent
   Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
      2017   -   NCT03045042   Spain
Filgrastim
   Serhat Gumrukcu, MD PhD
      2022   -   NCT04476550   United States
GC301
   GeneCradle Inc
      2024   Phase 1/Phase 2   NCT06391736   China
      2023   Phase 1/Phase 2   NCT05793307   China
   Seventh Medical Center of PLA General Hospital
      2022   -   NCT05567627   China
Genetic test: ACE polymorphism
   LMU Klinikum
      2022   -   NCT05272969   Germany
Genetic test: ACTN3 polymorphism
   LMU Klinikum
      2022   -   NCT05272969   Germany
GILT-rhgaa
   BioMarin Pharmaceutical Inc
      2016   Phase 3   EUCTR2013-001768-48-AT   Austria;Belgium;France;Germany;Italy;Japan;Netherlands;Portugal;Switzerland;United Kingdom;United States
      2015   Phase 3   EUCTR2013-001768-48-PT   Argentina;Austria;Belgium;Colombia;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States
      2014   Phase 3   EUCTR2013-001768-48-IT   Argentina;Austria;Belgium;Colombia;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States
      2014   Phase 3   EUCTR2013-001768-48-DE   Argentina;Austria;Belgium;Colombia;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States
      2014   Phase 3   EUCTR2013-001768-48-BE   Argentina;Austria;Belgium;Colombia;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States
      2013   Phase 3   EUCTR2013-001768-48-GB   Austria;Belgium;Colombia;Denmark;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States
      2013   Phase 2   EUCTR2011-001805-28-DE   Australia;Germany;New Zealand;United Kingdom;United States
      2012   Phase 2   EUCTR2011-001805-28-GB   Australia;France;Germany;United Kingdom;United States
      2012   -   EUCTR2010-023561-22-DE   France;Germany;Netherlands;United Kingdom;United States
      2011   Phase 1;Phase 2   EUCTR2010-023561-22-GB   Germany;United Kingdom;United States
Glucosidase alfa
   Klinikum der Universitaet Muenchen
      2016   -   NCT02824068   Argentina;Brazil;Germany;Italy;Taiwan
Glycosade
   John Mitchell
      2013   -   NCT02054832   Canada
GZ402666
      -   Phase 3   EUCTR2019-002251-42-NL   Netherlands
   GENZYME CORPORATION
      2016   Phase 3   EUCTR2016-000942-77-IT   Austria;Belgium;Czech Republic;Denmark;Germany;Italy;Netherlands;Poland;Spain;Sweden;United Kingdom
   Genzyme Corporation
      2019   Phase 3   EUCTR2016-000942-77-HU   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2016-000942-77-PT   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2016-000942-77-BG   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2017   Phase 2   EUCTR2016-003475-21-GB   France;Germany;Japan;Taiwan;United Kingdom;United States
      2017   Phase 3   EUCTR2016-000942-77-NL   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2016-000942-77-AT   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Türkiye;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-SE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-GB   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-ES   Argentina;Austria;Belgium;Brazil;Canada;Colombia;Czech Republic;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-DK   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-DE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-CZ   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-BE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2014   Phase 2   EUCTR2013-003321-28-NL   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States
      2014   -   EUCTR2013-003321-28-FR   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States
      2014   Phase 2   EUCTR2013-003321-28-DK   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States
      2014   Phase 2   EUCTR2013-003321-28-DE   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States
      2014   Phase 2   EUCTR2013-003321-28-BE   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States
      -   Phase 2   EUCTR2016-003475-21-FR   France;Germany;Japan;Taiwan;United Kingdom;United States
      -   Phase 2   EUCTR2016-003475-21-DE   France;Germany;Japan;Taiwan;United Kingdom;United States
      -   Phase 3   EUCTR2016-000942-77-PL   Argentina;Australia;Austria;Belgium;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
   Genzyme, a Sanofi Company
      2022   Phase 4   NCT05164055   France
      2017   Phase 2   NCT03019406   France;Japan;Taiwan;United Kingdom;United States
      2016   Phase 3   NCT02782741   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2013   Phase 1   NCT01898364   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States
   SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
      2021   Phase 3   EUCTR2020-004686-39-IT   Belgium;China;France;Germany;Italy;Netherlands;Spain;Taiwan;United Kingdom;United States
   Sanofi-Aventis Recherche & Développement
      2021   Phase 3   EUCTR2020-004686-39-NL   Belgium;China;France;Germany;Italy;Netherlands;Spain;Taiwan;United Kingdom;United States
      2021   Phase 3   EUCTR2020-004686-39-BE   Belgium;China;France;Germany;Italy;Netherlands;Spain;Taiwan;United Kingdom;United States
      -   Phase 4   EUCTR2021-002590-26-FR   France
      -   Phase 3   EUCTR2020-004686-39-DE   Belgium;China;France;Germany;Italy;Netherlands;Spain;Taiwan;United Kingdom;United States
GZ419829
   Genzyme Corporation
      -   Phase 4   EUCTR2021-005552-11-Outside-EU/EEA   United States
   Genzyme, a Sanofi Company
      2017   Phase 2   NCT03019406   France;Japan;Taiwan;United Kingdom;United States
      2016   Phase 3   NCT02782741   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
   Sanofi(China) Investment Co Ltd, Shanghai Branch
      -   Phase 4   EUCTR2021-004047-25-Outside-EU/EEA   China
Intravenous immune globulin
   National Taiwan University Hospital
      2015   Phase 4   NCT02525172   Taiwan
Laboratory assessment: calcium
   LMU Klinikum
      2022   -   NCT05272969   Germany
Laboratory assessment: creatine kinase
   LMU Klinikum
      2022   -   NCT05272969   Germany
Laboratory assessment: magnesium
   LMU Klinikum
      2022   -   NCT05272969   Germany
Laboratory assessment: phosphate
   LMU Klinikum
      2022   -   NCT05272969   Germany
Laboratory assessment: vitamin D level
   LMU Klinikum
      2022   -   NCT05272969   Germany
Laboratory tests
   Turkish Society of Pediatric Gastroenterology, Hepatology and Nutrition
      2019   -   NCT04120168   Turkey
Laronidase
   University of California, San Francisco
      2021   Phase 1   NCT04532047   United States
Lidocaine
   University of Florida
      2017   Phase 1   NCT02240407   United States
LMX 4 topical cream
   University of Florida
      2017   Phase 1   NCT02240407   United States
LOW carbohydrate ketogenic diet
   IRCCS Eugenio Medea
      2019   -   NCT04292938   Italy
Mannose
   Genzyme Corporation
      2019   Phase 3   EUCTR2016-000942-77-HU   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2016-000942-77-PT   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2016-000942-77-BG   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2017   Phase 2   EUCTR2016-003475-21-GB   France;Germany;Japan;Taiwan;United Kingdom;United States
      2017   Phase 3   EUCTR2016-000942-77-NL   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2016-000942-77-AT   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Türkiye;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-SE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-GB   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-ES   Argentina;Austria;Belgium;Brazil;Canada;Colombia;Czech Republic;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-DK   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-DE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-CZ   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-BE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      -   Phase 2   EUCTR2016-003475-21-FR   France;Germany;Japan;Taiwan;United Kingdom;United States
      -   Phase 2   EUCTR2016-003475-21-DE   France;Germany;Japan;Taiwan;United Kingdom;United States
      -   Phase 3   EUCTR2016-000942-77-PL   Argentina;Australia;Austria;Belgium;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
Methotrexate
   Genzyme, a Sanofi Company
      2009   Phase 4   NCT00701129   Israel;United States
   National Taiwan University Hospital
      2015   Phase 4   NCT02525172   Taiwan
Methotrexate sodium
   Genzyme Corporation Inc
      -   -   EUCTR2015-000584-14-Outside-EU/EEA   United States
      -   -   EUCTR2015-000583-34-Outside-EU/EEA   Israel;United States
Miglustat
   AMICUS THERAPEUTICS, INC.
      2020   Phase 3   EUCTR2019-000954-67-IT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Democratic People's Republic of;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-IT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Democratic People's Republic of;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
   Amicus Therapeutics
      2024   -   NCT06121011   Belgium;Germany;United Kingdom;United States
      2023   Phase 3   NCT04808505   France;Germany;Italy;Netherlands;Taiwan;United Kingdom;United States
      2020   Phase 3   NCT03911505   Australia;Canada;Germany;Italy;Japan;Taiwan;United States
   Amicus Therapeutics, Inc.
      2020   Phase 3   EUCTR2019-000954-67-SI   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-GR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-GB   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-FR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-ES   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-DK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-DE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-BE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2020   Phase 3   EUCTR2019-000954-67-AT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-SK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-SI   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-SE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-NL   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-HU   Australia;Austria;Belgium;Bosnia and Herzegovina;Brazil;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-GR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-GB   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-ES   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-DK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-DE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-BG   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-BE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-AT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2016   Phase 1;Phase 2   EUCTR2015-004798-34-NL   Australia;Germany;Netherlands;New Zealand;United Kingdom;United States
      2016   Phase 1;Phase 2   EUCTR2015-004798-34-GB   Australia;Germany;Netherlands;United Kingdom;United States
      2016   Phase 1;Phase 2   EUCTR2015-004798-34-DE   Australia;Germany;Netherlands;New Zealand;United Kingdom;United States
   Amicus Therapeutics, Inc./CMIC Co., Ltd.
      2019   Phase 3   JPRN-jRCT2080224804   Asia except Japan;Europe;Japan;North America;Oceania;South America
   UNIVERSITA` DEGLI STUDI DI NAPOLI FEDERICO II
      2011   -   EUCTR2010-024647-32-IT   Italy
   University of Florida
      2008   -   NCT01451879   United States
Muscle ultrasound
   LMU Klinikum
      2022   -   NCT05272969   Germany
Myozyme
      2007   Phase 4   EUCTR2007-001375-11-NL   Netherlands
   AMICUS THERAPEUTICS, INC.
      2019   Phase 3   EUCTR2018-000755-40-IT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Democratic People's Republic of;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
   Amicus Therapeutics, Inc.
      2019   Phase 3   EUCTR2018-000755-40-SK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-SI   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-SE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-NL   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-HU   Australia;Austria;Belgium;Bosnia and Herzegovina;Brazil;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-GR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-GB   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-ES   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-DK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-DE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-BG   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-BE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      2019   Phase 3   EUCTR2018-000755-40-AT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
      -   Phase 2   EUCTR2013-002257-30-GB   Netherlands;United Kingdom;United States
   GENZYME
      2005   -   EUCTR2005-001629-27-IT   Germany;Italy
      2004   -   EUCTR2004-002168-59-IT   Italy
   GENZYME CORPORATION
      2016   Phase 3   EUCTR2016-000942-77-IT   Austria;Belgium;Czech Republic;Denmark;Germany;Italy;Netherlands;Poland;Spain;Sweden;United Kingdom
   Genzyme
      -   Phase 4   EUCTR2024-000461-24-Outside-EU/EEA   China
   Genzyme Corporation
      2019   Phase 3   EUCTR2016-000942-77-HU   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2016-000942-77-PT   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2016-000942-77-BG   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2017   Phase 2   EUCTR2016-003475-21-GB   France;Germany;Japan;Taiwan;United Kingdom;United States
      2017   Phase 3   EUCTR2016-000942-77-NL   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2016-000942-77-AT   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Türkiye;United Kingdom;United States
      2017   Phase 4   EUCTR2010-022231-11-GB   Bulgaria;Germany;India;Russian Federation;Ukraine;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-SE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-GB   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-ES   Argentina;Austria;Belgium;Brazil;Canada;Colombia;Czech Republic;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-DK   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-DE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-CZ   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-BE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2012   Phase 1;Phase 4   EUCTR2010-022231-11-DE   Bulgaria;China;Germany;India;Poland;Russian Federation;Ukraine;United Kingdom;United States
      -   Phase 4   EUCTR2021-005552-11-Outside-EU/EEA   United States
      -   Phase 2   EUCTR2016-003475-21-FR   France;Germany;Japan;Taiwan;United Kingdom;United States
      -   Phase 2   EUCTR2016-003475-21-DE   France;Germany;Japan;Taiwan;United Kingdom;United States
      -   Phase 3   EUCTR2016-000942-77-PL   Argentina;Australia;Austria;Belgium;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
   Genzyme Corporation Inc
      -   -   EUCTR2015-000584-14-Outside-EU/EEA   United States
      -   -   EUCTR2015-000583-34-Outside-EU/EEA   Israel;United States
   Genzyme Corporation, a Sanofi company
      2013   Phase 4   EUCTR2011-005595-42-DE   Canada;Germany;India;Russian Federation;Taiwan;Turkey;United States
   Genzyme Europe B.V.
      2012   -   EUCTR2010-020611-36-NL   Denmark;Germany;Netherlands;United Kingdom;United States
      2011   -   EUCTR2010-020611-36-GB   Germany;Netherlands;United Kingdom;United States
      2011   -   EUCTR2010-020611-36-DE   Germany;Netherlands;United Kingdom;United States
      2007   -   EUCTR2006-003644-31-DE   France;Germany;Netherlands
   Genzyme Europe BV
      2007   -   EUCTR2006-003644-31-NL   France;Germany;Netherlands
      2007   Phase 4   EUCTR2006-003644-31-FR   France;Germany;Netherlands
      2007   -   EUCTR2005-002759-42-DE   Germany
      2005   Phase 2   EUCTR2005-002829-31-GB   Belgium;United Kingdom
      2005   -   EUCTR2005-001629-27-DE   Germany;Italy
      -   Phase 2   EUCTR2005-002829-31-BE   Belgium;United Kingdom
   Genzyme, a Sanofi Company
      2018   Phase 4   NCT03687333   China
      2008   Phase 4   NCT00701701   Canada;Israel;United States
      2005   Phase 3   NCT00268944   France
      2005   Phase 2   NCT00250939   Netherlands
      2005   Phase 2/Phase 3   NCT00125879   France;Germany;Israel;Italy;Netherlands;Taiwan;United States
      2004   -   NCT00074932   United States
      2003   Phase 2   NCT00765414   United States
      2003   Phase 2   NCT00763932   France;South Africa;United States
      2003   Phase 2/Phase 3   NCT00059280   France;Israel;Taiwan;United Kingdom;United States
      2003   Phase 1/Phase 2   NCT00053573   France;Israel;United Kingdom;United States
   Sanofi(China) Investment Co Ltd, Shanghai Branch
      -   Phase 4   EUCTR2021-004047-25-Outside-EU/EEA   China
   Valerion Therapeutics, LLC
      2017   Phase 1;Phase 2   EUCTR2016-004578-16-GB   United Kingdom;United States
Myozyme 50 MG powder FOR concentrate FOR solution FOR infusion
   Genzyme Corporation Inc
      -   -   EUCTR2015-000582-31-Outside-EU/EEA   Australia;Canada;United States
NEO-GAA
   Genzyme Corporation
      2017   Phase 3   EUCTR2016-000942-77-NL   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
Neogaa
   GENZYME CORPORATION
      2016   Phase 3   EUCTR2016-000942-77-IT   Austria;Belgium;Czech Republic;Denmark;Germany;Italy;Netherlands;Poland;Spain;Sweden;United Kingdom
   Genzyme Corporation
      2019   Phase 3   EUCTR2016-000942-77-HU   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2016-000942-77-BG   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2017   Phase 2   EUCTR2016-003475-21-GB   France;Germany;Japan;Taiwan;United Kingdom;United States
      2017   Phase 3   EUCTR2016-000942-77-NL   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-SE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-ES   Argentina;Austria;Belgium;Brazil;Canada;Colombia;Czech Republic;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-DK   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-CZ   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-BE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2014   Phase 2   EUCTR2013-003321-28-NL   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States
      2014   -   EUCTR2013-003321-28-FR   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States
      -   Phase 2   EUCTR2016-003475-21-FR   France;Germany;Japan;Taiwan;United Kingdom;United States
      -   Phase 2   EUCTR2016-003475-21-DE   France;Germany;Japan;Taiwan;United Kingdom;United States
Neogaa or avalglucosidase alfa
   Genzyme Corporation
      2018   Phase 3   EUCTR2016-000942-77-PT   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2016-000942-77-AT   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Türkiye;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-GB   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-DE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      -   Phase 3   EUCTR2016-000942-77-PL   Argentina;Australia;Austria;Belgium;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
Neutralizing antibody TO SPK-3006 capsid
   Spark Therapeutics
      2019   -   NCT03893240   France;Germany;Italy;Netherlands;United Kingdom;United States
Nexviadyme
   Sanofi-Aventis Recherche & Développement
      2021   Phase 3   EUCTR2020-004686-39-NL   Belgium;China;France;Germany;Italy;Netherlands;Spain;Taiwan;United Kingdom;United States
      2021   Phase 3   EUCTR2020-004686-39-BE   Belgium;China;France;Germany;Italy;Netherlands;Spain;Taiwan;United Kingdom;United States
      -   Phase 3   EUCTR2020-004686-39-DE   Belgium;China;France;Germany;Italy;Netherlands;Spain;Taiwan;United Kingdom;United States
NON applicabile
   AMICUS THERAPEUTICS, INC.
      2019   Phase 3   EUCTR2018-000755-40-IT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Democratic People's Republic of;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States
Phosphate
   Genzyme Corporation
      2019   Phase 3   EUCTR2016-000942-77-HU   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2016-000942-77-PT   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2018   Phase 3   EUCTR2016-000942-77-BG   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2017   Phase 2   EUCTR2016-003475-21-GB   France;Germany;Japan;Taiwan;United Kingdom;United States
      2017   Phase 3   EUCTR2016-000942-77-NL   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2017   Phase 3   EUCTR2016-000942-77-AT   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Türkiye;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-SE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-GB   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-ES   Argentina;Austria;Belgium;Brazil;Canada;Colombia;Czech Republic;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-DK   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-DE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-CZ   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      2016   Phase 3   EUCTR2016-000942-77-BE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
      -   Phase 2   EUCTR2016-003475-21-FR   France;Germany;Japan;Taiwan;United Kingdom;United States
      -   Phase 2   EUCTR2016-003475-21-DE   France;Germany;Japan;Taiwan;United Kingdom;United States
      -   Phase 3   EUCTR2016-000942-77-PL   Argentina;Australia;Austria;Belgium;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States
Pressure pain threshold
   LMU Klinikum
      2022   -   NCT05272969   Germany
Pyridostigmine bromide
   University of Florida
      2015   Early Phase 1   NCT02357225   United States
Quick motor function test
   LMU Klinikum
      2022   -   NCT05272969   Germany
RAAV1-CMV-GAA (study agent) administration
   University of Florida
      2010   Phase 1/Phase 2   NCT00976352   United States
Rapamycin
   University of Florida
      2017   Phase 1   NCT02240407   United States
Recombinant adeno-associated virus acid alpha-glucosidase
   University of Florida
      2017   Phase 1   NCT02240407   United States
Recombinant human ?-glucosidase conjugated with synthetic bismannose-6-phosphate-MAN6 glycan
   Genzyme Corporation
      2014   Phase 2   EUCTR2013-003321-28-NL   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States
      2014   -   EUCTR2013-003321-28-FR   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States
      2014   Phase 2   EUCTR2013-003321-28-DK   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States
      2014   Phase 2   EUCTR2013-003321-28-DE   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States
      2014   Phase 2   EUCTR2013-003321-28-BE   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States
   Sanofi-Aventis Recherche & Développement
      2021   Phase 3   EUCTR2020-004686-39-NL   Belgium;China;France;Germany;Italy;Netherlands;Spain;Taiwan;United Kingdom;United States
      2021   Phase 3   EUCTR2020-004686-39-BE   Belgium;China;France;Germany;Italy;Netherlands;Spain;Taiwan;United Kingdom;United States
      -   Phase 4   EUCTR2021-002590-26-FR   France
      -   Phase 3   EUCTR2020-004686-39-DE   Belgium;China;France;Germany;Italy;Netherlands;Spain;Taiwan;United Kingdom;United States
Recombinant human ALFA-glucosidase conjugated with synthetic bismannose-6-phosphate-MAN6 glycan
   SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
      2021   Phase 3   EUCTR2020-004686-39-IT   Belgium;China;France;Germany;Italy;Netherlands;Spain;Taiwan;United Kingdom;United States
REN001
   Reneo Pharma Ltd
      2019   Phase 1   NCT04226274   Spain;United Kingdom
Reveglucosidase alfa
   BioMarin Pharmaceutical Inc
      2013   Phase 3   EUCTR2013-001768-48-GB   Austria;Belgium;Colombia;Denmark;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States
Reveglucosidase alpha
   BioMarin Pharmaceutical Inc
      2016   Phase 3   EUCTR2013-001768-48-AT   Austria;Belgium;France;Germany;Italy;Japan;Netherlands;Portugal;Switzerland;United Kingdom;United States
      2014   Phase 3   EUCTR2013-001768-48-DE   Argentina;Austria;Belgium;Colombia;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States
      2014   Phase 3   EUCTR2013-001768-48-BE   Argentina;Austria;Belgium;Colombia;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States
Rhgaa
   Amicus Therapeutics
      2011   Phase 2   NCT01380743   Canada;France;United Kingdom;United States
   Erasmus MC Sophia
      2009   -   EUCTR2008-008293-31-NL   Netherlands
   GENZYME
      2004   -   EUCTR2004-002168-59-IT   Italy
   Genzyme Corporation
      2017   Phase 4   EUCTR2010-022231-11-GB   Bulgaria;Germany;India;Russian Federation;Ukraine;United Kingdom;United States
      2012   Phase 1;Phase 4   EUCTR2010-022231-11-DE   Bulgaria;China;Germany;India;Poland;Russian Federation;Ukraine;United Kingdom;United States
   Genzyme Corporation Inc
      -   -   EUCTR2015-000512-18-Outside-EU/EEA   United States
   Genzyme Corporation, a Sanofi company
      2013   Phase 4   EUCTR2011-005595-42-DE   Canada;Germany;India;Russian Federation;Taiwan;Turkey;United States
   Genzyme Europe B.V.
      2012   -   EUCTR2010-020611-36-NL   Denmark;Germany;Netherlands;United Kingdom;United States
      2011   -   EUCTR2010-020611-36-GB   Germany;Netherlands;United Kingdom;United States
      2011   -   EUCTR2010-020611-36-DE   Germany;Netherlands;United Kingdom;United States
   Genzyme Europe BV
      2007   -   EUCTR2005-002759-42-DE   Germany
      2005   -   EUCTR2005-001629-27-DE   Germany;Italy
   Genzyme, a Sanofi Company
      2007   -   NCT00520143   United States
      2001   Phase 2   NCT00025896   United States
   Valerion Therapeutics, LLC
      2017   Phase 1/Phase 2   NCT02898753   United Kingdom;United States
      2017   Phase 1;Phase 2   EUCTR2016-004578-16-GB   United Kingdom;United States
Rhgaa, acid alpha glucosidase
   Genzyme Europe B.V.
      2007   -   EUCTR2006-003644-31-DE   France;Germany;Netherlands
   Genzyme Europe BV
      2007   -   EUCTR2006-003644-31-NL   France;Germany;Netherlands
      2007   Phase 4   EUCTR2006-003644-31-FR   France;Germany;Netherlands
      2005   Phase 2   EUCTR2005-002829-31-GB   Belgium;United Kingdom
      -   Phase 2   EUCTR2005-002829-31-BE   Belgium;United Kingdom
Rituxan
   University of Florida
      2017   Phase 1   NCT02240407   United States
Rituximab
   Genzyme Corporation Inc
      -   -   EUCTR2015-000584-14-Outside-EU/EEA   United States
      -   -   EUCTR2015-000583-34-Outside-EU/EEA   Israel;United States
   Genzyme, a Sanofi Company
      2009   Phase 4   NCT00701129   Israel;United States
   National Taiwan University Hospital
      2015   Phase 4   NCT02525172   Taiwan
   University of Florida
      2008   -   NCT01451879   United States
Rmst
   University of Florida
      2010   Phase 1/Phase 2   NCT00976352   United States
Salbutamol
   National Taiwan University Hospital
      2013   Phase 4   NCT02405598   Taiwan
Saline
   University of Florida
      2017   Phase 1   NCT02240407   United States
Sodium valproate
   John Vissing
      2014   Phase 2   EUCTR2014-001637-88-DK   Denmark
   University College, London
      2015   Phase 2   NCT03112889   Denmark;United Kingdom
SPK-3006
   Spark Therapeutics
      2021   Phase 1;Phase 2   EUCTR2019-001283-30-NL   France;Germany;Italy;Netherlands;United Kingdom;United States
      2021   Phase 1;Phase 2   EUCTR2019-001283-30-DK   Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
      2020   Phase 1;Phase 2   EUCTR2019-001283-30-FR   Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States
      2020   Phase 1;Phase 2   EUCTR2019-001283-30-DE   Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
   Spark Therapeutics, Inc.
      2020   Phase 1/Phase 2   NCT04093349   Canada;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States
Triheptanoin
   Copenhagen Neuromuscular Center
      2018   Phase 2   EUCTR2017-004153-17-DK   Denmark
   Institut National de la Santé Et de la Recherche Médicale, France
      2016   Phase 2   NCT02919631   France
   Neuromuscular Research Unit, Rigshospitalet
      2015   Phase 2   EUCTR2014-003644-12-DK   Denmark
   Rigshospitalet, Denmark
      2018   Phase 2   NCT03642860   Denmark
      2015   Phase 2   NCT02432768   Denmark
UX007
   Copenhagen Neuromuscular Center
      2018   Phase 2   EUCTR2017-004153-17-DK   Denmark
   Neuromuscular Research Unit, Rigshospitalet
      2015   Phase 2   EUCTR2014-003644-12-DK   Denmark
VAL-1221
   Valerion Therapeutics, LLC
      2017   Phase 1/Phase 2   NCT02898753   United Kingdom;United States
      2017   Phase 1;Phase 2   EUCTR2016-004578-16-GB   United Kingdom;United States
Vital signs
   LMU Klinikum
      2022   -   NCT05272969   Germany
Zavesca
   UNIVERSITA` DEGLI STUDI DI NAPOLI FEDERICO II
      2011   -   EUCTR2010-024647-32-IT   Italy
Zavesca® prescription
   University of Florida
      2016   Phase 1   NCT02185651   United States
Zocaglusagene nuzaparvovec
   Astellas Gene Therapies
      2020   Phase 1/Phase 2   NCT04174105   Germany;United Kingdom;United States