Glycogen Storage Disease Type II - 5 Studies Found
Completed |
: A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease :
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Completed |
: An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease :
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Recruiting |
: NeoGAA Extension Study : Glycogen Storage Disease Type II Pompe Disease : 2013-12-04 : Drug: GZ402666 Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration |
Recruiting |
: Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease :
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RECRUITING |
: A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 : Glycogen Storage Disease Type II Infantile Onset : 2024-11-21 : Sterile lyophilized powder intravenous (IV) infusion |