Angelman syndrome

Search Trials
A Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome
Status:
Recruiting
Last Changed:
Jun 8, 2022
First Changed:
Jun 11, 2020
Disease(s):
Angelman Syndrome
Intervention(s):
RO7248824
Angelman Syndrome Natural History Study
Status:
Recruiting
Last Changed:
Aug 4, 2021
First Changed:
Aug 11, 2020
Disease(s):
Angelman Syndrome
A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)
Status:
Recruiting
Last Changed:
Dec 21, 2021
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Withdrawn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
Study of Phenotype and Genotype Correlations in Patients With Contiguous Gene Deletion Syndromes
Status:
Completed
Last Changed:
Jun 24, 2005
First Changed:
Oct 19, 1999
Disease(s):
Williams Syndrome

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