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Glycogen Storage Disease Type II - 32 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:
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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 |
Recruiting |
Nombre del estudio: Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease Condición:
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Terminated |
Nombre del estudio: Study to Evaluate the Safety of AT2220 in Pompe Disease Condición: Pompe Disease Fecha: 2008-05-30 Intervenciones:
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Completed |
Nombre del estudio: Safety and Efficacy of Clenbuterol on Motor Function in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy Condición: Pompe Disease Fecha: 2013-09-11 Intervenciones:
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Recruiting |
Nombre del estudio: A Long Term Follow up Study in Late-onset Pompe Disease Condición: Pompe Disease Fecha: 2008-07-09 |
Recruiting |
Nombre del estudio: A Pilot Study of Pyridostigmine in Pompe Disease Condición: Pompe Disease Fecha: 2015-01-23 Intervenciones: Drug: Pyridostigmine Bromide Pyridostigmine is an acetylcholinesterase inhibitor, which increases the am |