Aromatic Amino Acid Decarboxylase Deficiency

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Genetic Etiology in Patients With Cerebral Palsy
Status:
Enrolling by invitation
Last Changed:
Nov 24, 2021
First Changed:
Nov 17, 2021
Disease(s):
Cerebral Palsy
Intervention(s):
NGS
A Study of SmartFlow® Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants
Status:
Recruiting
Last Changed:
Jan 11, 2022
First Changed:
May 26, 2021
Disease(s):
AADC Deficiency
Intervention(s):
Eladocagene Exuparvovec
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Withdrawn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
A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC
Status:
Active, not recruiting
Last Changed:
Aug 11, 2021
First Changed:
Jul 15, 2011
Disease(s):
Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
Intervention(s):
gene therapy

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