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Glycogen Storage Disease Type I - 42 Studies Found
Estado | Estudiar |
Recruiting |
Nombre del estudio: Pompe Disease Registry Condición:
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Completed |
Nombre del estudio: Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease Condición:
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Approved for marketing |
Nombre del estudio: Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease Condición:
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Completed |
Nombre del estudio: 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 Condición:
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Completed |
Nombre del estudio: 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. Condición:
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Completed |
Nombre del estudio: A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease Condición:
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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:
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Completed |
Nombre del estudio: A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II Condición:
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Recruiting |
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: Newborn Screening Assay of Pompe's Disease Condición: Pompe Disease Fecha: 2008-07-09 Intervenciones: Other: Pompe disease newborn screening DBS will be tested for acid alpha-glucosidase (GAA)activity. Newb |