Glycogen Storage Disease Type I - 6 Studies Found

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

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
:
  • Pompe Disease (Late-Onset)
  • Glycogen Stora
    : 2011-08-02
    : Biological: alglucosidase alfa Intravenous (IV) infusion of 20mg/kg body weight every other week (qow)
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
RECRUITING : Prospective Collection of Biospecimen in Pediatric Patients and Adult Guardians Diagnosed With Glycogen Storage Disease Type 1B (GSD1b)
: Glycogen Storage Disease Type IB
: 2024-11-21
: blood donation