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Found 44 studies with search of: Pompe Disease
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Rank Status Study
1 Recruiting Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease
Condition: Pompe Disease
Intervention: Drug: rAAV1-CMV-GAA
2 Completed
Has Results 		A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease
Conditions: Pompe Disease (Late-onset);   Glycogen Storage Disease Type II (GSD-II);   Acid Maltase Deficiency Disease;   Glycogenosis 2
Interventions: Biological: alglucosidase alfa;   Drug: Placebo
3 Completed Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease
Condition: Pompe Disease
Intervention:
4 Completed Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease
Conditions: Pompe Disease;   Glycogen Storage Disease Type II;   Acid Maltase Deficiency Disease;   Glycogenosis 2
Intervention: Drug: recombinant human acid alpha-glucosidase (rhGAA)
5 Recruiting Pompe Pregnancy Sub-Registry
Conditions: Glycogen Storage Disease Type II (GSD-II);   Pompe Disease (Late-onset);   Glycogenesis 2 Acid Maltase Deficiency
Intervention:
6 Completed Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease
Conditions: Glycogen Storage Disease Type II;   Glycogenosis 2
Intervention: Biological: Myozyme
7 Completed A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II
Conditions: Glycogen Storage Disease Type II;   Pompe Disease;   Acid Maltase Deficiency Disease;   Glycogenosis 2
Intervention: Drug: Alglucosidase alfa
8 Completed rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)
Conditions: Glycogen Storage Disease Type II;   Pompe Disease;   Acid Maltase Deficiency Disease;   Glycogenosis 2
Intervention: Biological: Myozyme
9 Completed A Study of rhGAA in Patients With Late-Onset Pompe Disease
Conditions: Pompe Disease (Late-Onset);   Glycogen Storage Disease Type II (GSD-II);   Acid Maltase Deficiency Disease;   Glycogenosis 2
Intervention: Biological: Myozyme
10 Completed Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support
Conditions: Pompe Disease (Late-Onset);   Glycogen Storage Disease Type II (GSD-II);   Acid Maltase Deficiency Disease;   Glycogenosis 2
Intervention: Biological: Myozyme
11 Completed A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease
Conditions: Pompe Disease;   Glycogen Storage Disease Type II
Intervention: Other: Observation
12 Recruiting Pompe Disease Registry
Conditions: Glycogen Storage Disease Type II;   Pompe Disease
Intervention:
13 Unknown † Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease
Conditions: Glycogen Storage Disease Type II;   Glycogenosis 2
Intervention: Biological: alglucosidase alfa
14 Completed A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-Onset Pompe Disease
Condition: Glycogen Storage Disease Type II
Intervention: Biological: Myozyme
15 Completed Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®
Conditions: Pompe Disease (Late-Onset);   Glycogen Storage Disease Type II (GSD II);   Glycogenesis 2 Acid Maltase Deficiency
Intervention:
16 Recruiting Pompe Lactation Sub-Registry
Conditions: Glycogen Storage Disease;   Pompe Disease
Intervention:
17 Completed A Prospective, Observational Study in Patients With Late-Onset Pompe Disease
Condition: Glycogen Storage Disease Type II
Intervention:
18 Terminated Study to Evaluate the Safety of AT2220 in Pompe Disease
Condition: Pompe Disease
Intervention: Drug: AT2220
19 Recruiting Observational Study for Children With Pompe Disease Undergoing Immune Modulation Therapies
Condition: Pompe Disease
Intervention:
20 Recruiting Biomarker for Pompe Disease
Conditions: Lysosomal Storage Diseases;   Pompe Disease
Intervention:

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