Glycogen Storage Disease Type 4

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Withdrawn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
Second-line Therapy of Unresectable Cholangiocarcinoma by RADIOEMBOLIZATION
Status:
Terminated
Last Changed:
Aug 7, 2013
First Changed:
Jun 28, 2011
Disease(s):
Cholestasis, Progressive Familial Intrahepatic 3
Intervention(s):
Yttrium microsphere injection
Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa
Status:
Completed
Last Changed:
Dec 19, 2014
First Changed:
Feb 2, 2011
Disease(s):
Pompe Disease (Late-Onset)
Intervention(s):
Alglucosidase Alfa
A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease
Status:
Terminated
Last Changed:
Dec 8, 2015
First Changed:
Feb 6, 2012
Disease(s):
Pompe Disease
Intervention(s):
Alglucosidase alfa

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