Glycogen Storage Disease Type I - 42 Studies Found
Recruiting |
: Pompe Disease Registry :
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Completed |
: Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease :
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Approved for marketing |
: Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease :
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Completed |
: Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies :
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Completed |
: Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies. :
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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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Completed |
: A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II :
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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 |
Completed |
: Newborn Screening Assay of Pompe's Disease : Pompe Disease : 2008-07-09 : Other: Pompe disease newborn screening DBS will be tested for acid alpha-glucosidase (GAA)activity. Newb |