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DR info

Clinical trials : 13 / Drugs : 37 - (DrugBank : 7) / Drug target genes : 2 - Drug target pathways : 53

Drugs, Primary sponsors and Trial info (Enrollment year, Phase, Trial ID, Countries)

Apotamox 10MG
   James Dowling
      2020   Phase 1/Phase 2   NCT04915846   Canada;United Kingdom;United States
ASP2957
   Astellas Gene Therapies
      2025   Phase 1/Phase 2   NCT07052929   Canada;United States
AT132
   Astellas Gene Therapies, Inc.
      2018   Phase 1;Phase 2;Phase 3   EUCTR2017-000876-27-DE   Canada;France;Germany;United Kingdom;United States
   Audentes Therapeutics Inc.
      2018   Phase 1;Phase 2   EUCTR2017-000876-27-FR   France;Germany;United Kingdom;United States
      2017   Phase 1;Phase 2   EUCTR2017-000876-27-GB   France;Germany;United Kingdom;United States
Blood sample collection
   Centre Hospitalier Universitaire de Liege
      2026   -   NCT07201636   Belgium
Bulbar function questionnaires
   Centre Hospitalier Universitaire de Liege
      2026   -   NCT07201636   Belgium
Complete physical examination
   Hospital Universitari Vall d'Hebron Research Institute
      2026   -   NCT07488806   Spain
Digital biomarkers
   Hospital Universitari Vall d'Hebron Research Institute
      2026   -   NCT07488806   Spain
DYN101
   Dynacure
      -   Phase 1;Phase 2   EUCTR2020-004608-32-DE   France;Germany;Netherlands;Spain
L-tyrosine
   Hadassah Medical Organization
      2014   Phase 2   NCT02035501   Israel
Methylprednisolone
   Astellas Gene Therapies
      2025   Phase 1/Phase 2   NCT07052929   Canada;United States
Motor function assessments
   Centre Hospitalier Universitaire de Liege
      2026   -   NCT07201636   Belgium
Motor function scales
   Hospital Universitari Vall d'Hebron Research Institute
      2026   -   NCT07488806   Spain
Motor milestone assessments
   Hospital Universitari Vall d'Hebron Research Institute
      2026   -   NCT07488806   Spain
Muscle ultrasound
   Hospital Universitari Vall d'Hebron Research Institute
      2026   -   NCT07488806   Spain
N-acetylcysteine
   National Institute of Nursing Research (NINR)
      2015   Phase 1/Phase 2   NCT02362425   United States
Oromotor function and nutrition
   Hospital Universitari Vall d'Hebron Research Institute
      2026   -   NCT07488806   Spain
Prednisolone
   Astellas Gene Therapies
      2025   Phase 1/Phase 2   NCT07052929   Canada;United States
QOL assessment, neuropsychological
   Hospital Universitari Vall d'Hebron Research Institute
      2026   -   NCT07488806   Spain
Quality OF life questionnaires
   Centre Hospitalier Universitaire de Liege
      2026   -   NCT07201636   Belgium
RAAV8-DES-HMTM1
   Astellas Gene Therapies, Inc.
      2018   Phase 1;Phase 2;Phase 3   EUCTR2017-000876-27-DE   Canada;France;Germany;United Kingdom;United States
   Audentes Therapeutics Inc.
      2018   Phase 1;Phase 2   EUCTR2017-000876-27-FR   France;Germany;United Kingdom;United States
      2017   Phase 1;Phase 2   EUCTR2017-000876-27-GB   France;Germany;United Kingdom;United States
Resamirigene bilparvovec
   Astellas Gene Therapies
      2017   Phase 2/Phase 3   NCT03199469   Canada;France;Germany;United States
Respiratory assessments
   Centre Hospitalier Universitaire de Liege
      2026   -   NCT07201636   Belgium
Salbutamol (AS salbutamol sulfate) 2 MG oral tablet
   Vastra Gotaland Region
      2021   -   NCT05099107   Sweden
Salbutamol 2MG
   Västra götalandsregionen
      2020   Phase 4   EUCTR2019-001147-51-SE   Sweden
Salbutamol 4MG
   Västra götalandsregionen
      2020   Phase 4   EUCTR2019-001147-51-SE   Sweden
Salbutamol only product IN oral dose form
   Vastra Gotaland Region
      2021   -   NCT05099107   Sweden
Salbutamol sulfate
   Västra götalandsregionen
      2020   Phase 4   EUCTR2019-001147-51-SE   Sweden
Sirolimus
   Astellas Gene Therapies
      2025   Phase 1/Phase 2   NCT07052929   Canada;United States
Syde
   Centre Hospitalier Universitaire de Liege
      2026   -   NCT07201636   Belgium
Ventilatory/ respiratory, cardiac and other support assessment
   Hospital Universitari Vall d'Hebron Research Institute
      2026   -   NCT07488806   Spain
Ventoline oral solution 0,4MG/ML
   Västra götalandsregionen
      2020   Phase 4   EUCTR2019-001147-51-SE   Sweden
Ventoline tablet 2MG
   Västra götalandsregionen
      2020   Phase 4   EUCTR2019-001147-51-SE   Sweden