Leukodystrophy

Search Trials
A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)
Status:
Recruiting
Last Changed:
Aug 7, 2019
First Changed:
Apr 5, 2019
Disease(s):
Progressive Familial Intrahepatic Cholestasis (PFIC)
Intervention(s):
MaralixibatPlacebo
A Placebo-controlled Study of Maralixibat (SHP625) in Pediatric Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)
Status:
Withdrawn
Last Changed:
Mar 18, 2019
First Changed:
Nov 27, 2017
Disease(s):
Progressive Familial Intrahepatic Cholestasis (PFIC)
Intervention(s):
MaralixibatPlacebo
A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)
Status:
Recruiting
Last Changed:
Dec 19, 2018
First Changed:
Jan 8, 2018
Disease(s):
Lysosomal Storage Disease
Intervention(s):
OTL-200
Gene Therapy for Metachromatic Leukodystrophy (MLD)
Status:
Active, not recruiting
Last Changed:
Dec 19, 2018
First Changed:
Mar 22, 2012
Disease(s):
Lysosomal Storage Disease
Intervention(s):
OTL-200 Gene Therapy
A Study of Intrathecal SHP611 in Participants With Late Infantile Metachromatic Leukodystrophy
Status:
Not yet recruiting
Last Changed:
Dec 11, 2018
First Changed:
Dec 11, 2018
Disease(s):
Metachromatic Leukodystrophy (MLD)
Intervention(s):
SHP611

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