Glycogen Storage Disease Type 4

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GBE Deficiency (GSD IV and APBD) Natural History Study
Status:
Recruiting
Last Changed:
Dec 10, 2021
First Changed:
Feb 17, 2016
Disease(s):
Glycogen Storage Disease Type IV
Intervention(s):
No intervention
Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)
Status:
Not yet recruiting
Last Changed:
Mar 11, 2022
First Changed:
Dec 20, 2021
Disease(s):
Glycogen Storage Disease Type II
Intervention(s):
Avalglucosidase alfa (GZ402666)
Rare Disease Patient Registry & Natural History Study – Coordination of Rare Diseases at Sanford
Status:
Recruiting
Last Changed:
Dec 15, 2021
First Changed:
Feb 15, 2013
Disease(s):
Rare Disorders
Biomarker for Glycogen Storage Diseases (BioGlycogen)
Status:
Active, not recruiting
Last Changed:
May 13, 2021
First Changed:
Mar 11, 2015
Disease(s):
Fructose Metabolism, Inborn Errors
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

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