Roussy Levy syndrome

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Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)
Status:
Recruiting
Last Changed:
Aug 28, 2019
First Changed:
May 9, 2019
Disease(s):
Facioscapulohumeral Muscular Dystrophy
Intervention(s):
ACE-083
A Registered Cohort Study on Charcot-Marie-Tooth Disease
Status:
Recruiting
Last Changed:
Jul 16, 2019
First Changed:
Jul 8, 2019
Disease(s):
Charcot-Marie-Tooth Disease
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Withdrawn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 Natural History Study of Charcot-Marie-Tooth 4J (CMT4J)
Status:
Recruiting
Last Changed:
Jul 30, 2019
First Changed:
Jan 18, 2019
Disease(s):
Charcot-Marie-Tooth Disease

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