Cornelia de Lange Syndrome (CdLS)

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Rare Disease Patient Registry & Natural History Study – Coordination of Rare Diseases at Sanford
Status:
Recruiting
Last Changed:
Dec 10, 2020
First Changed:
Feb 15, 2013
Disease(s):
Rare Disorders
Use of N-Acetylcysteine in the Treatment of Repetitive and Self-Injurious Behaviors in Cornelia de Lange Syndrome
Status:
Not yet recruiting
Last Changed:
Sep 17, 2021
First Changed:
May 11, 2020
Disease(s):
Cornelia de Lange Syndrome
Intervention(s):
N-acetyl cysteinePlacebo
GROWing Up With Rare GENEtic Syndromes
Status:
Recruiting
Last Changed:
Jul 9, 2020
First Changed:
Jul 9, 2020
Disease(s):
Prader-Willi Syndrome
Intervention(s):
Retrospective file studies
A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)
Status:
Recruiting
Last Changed:
Sep 14, 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Withdrawn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

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