Glycogen Storage Disease Type Ii
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CLINICAL_TRIALS research · 2026-04-09Clinical Trial Update: Pompe Disease Registry Protocol
This entry describes a Pompe disease registry protocol sponsored by Genzyme (a Sanofi company). The study is currently recruiting.
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CLINICAL_TRIALS research · 2026-01-22Clinical Trial Update: A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 [Phase 3]
Phase 3 trial to evaluate the safety, efficacy, pharmacokinetics, pharmacodynamics and immunogenicity of cipaglucosidase alfa with miglustat…
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CLINICAL_TRIALS research · 2025-08-11Clinical Trial Update: Carbon-13 Magnetic Resonance Spectroscopy in Glycogen Storage Diseases
This update describes a clinical study using carbon-13 magnetic resonance spectroscopy to investigate glycogen storage diseases, including M…
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CLINICAL_TRIALS research · 2016-03-15Clinical Trial Update: An MRI Study on Muscular Diseases -Pompe Disease and Dystrophia Myotonica-
An MRI-focused clinical study update investigating muscular diseases, specifically Pompe disease (glycogen storage disease type II) and myot…
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CLINICAL_TRIALS research · 2008-06-05Clinical Trial Update: A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease
A completed study evaluated the effects of pharmacological chaperones on cells derived from patients with Pompe disease.
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CLINICAL_TRIALS research · 2008-03-05Clinical Trial Update: Genetic and Family Studies of Inherited Muscle Diseases
NIAMS-sponsored clinical trial investigating genetic and familial factors in inherited muscle diseases has completed, involving conditions s…