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Glycogen Storage Disease Type II - 5 Studies Found

Estado Estudiar
Completed Nombre del estudio: A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease
Condición:
  • Pompe Disease
  • Glycogen Storage Disease Ty
    Fecha: 2007-08-10
    Intervenciones: Other: Observation Ex vivo administration of AT2220
Completed Nombre del estudio: 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
Condición:
  • Pompe Disease
  • Glycogen Storage Disease Ty
    Fecha: 2008-06-17
    Intervenciones:
    • Biological: Alglucosidase Alfa

Completed Nombre del estudio: NeoGAA Extension Study
Condición: Glycogen Storage Disease Type II Pompe Disease
Fecha: 2013-12-04
Intervenciones: Drug: GZ402666 Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration
Completed Nombre del estudio: Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease
Condición:
  • Pompe Disease (Late-Onset)
  • Glycogen Stora
    Fecha: 2011-08-02
    Intervenciones: Biological: alglucosidase alfa Intravenous (IV) infusion of 20mg/kg body weight every other week (qow)
Completed Nombre del estudio: A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
Condición: Glycogen Storage Disease Type II Infantile Onset
Fecha: 2025-11-04
Intervenciones: Sterile lyophilized powder intravenous (IV) infusion