DDrare
Disease
Drug
Target Gene/Pathway
問合せ先
EN
65. 原発性免疫不全症候群
Primary immunodeficiency
Clinical trials
/
Disease details
臨床試験数
:
500
/
薬物数
:
614
- (
DrugBank
:
119
) /
標的遺伝子数
:
92
-
標的パスウェイ数
:
217
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1
NCT03333200
(
ClinicalTrials.gov
)
January 11, 2012
24/4/2017
Longitudinal Study of Neurodegenerative Disorders
Longitudinal Study of Neurodegenerative Disorders
MLD;Krabbe Disease;ALD;MPS I;MPS II;MPS III;Vanishing White Matter Disease;GM3 Gangliosidosis;PKAN;Tay-Sachs Disease;NP Deficiency;Osteopetrosis;Alpha-Mannosidosis;Sandhoff Disease;Niemann-Pick Diseases;MPS IV;Gaucher Disease;GAN;GM1 Gangliosidoses;Morquio Disease;S-Adenosylhomocysteine Hydrolase Deficiency;Batten Disease;Pelizaeus-Merzbacher Disease;Leukodystrophy;Lysosomal Storage Diseases;Purine Nucleoside Phosphorylase Deficiency;Multiple Sulfatase Deficiency Disease
MLD;Krabbe Disease;ALD;MPS I;MPS II;MPS III;Vanishing White Matter Disease;GM3 Gangliosidosis;PKAN;T ...
Other: Palliative Care;Biological:
Hematopoetic Stem Cell Transplantation
University of Pittsburgh
NULL
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1500
United States