84 studies found for:    "Glycogen storage disease type 2"
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Rank Status Study
1 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
2 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
3 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
4 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
5 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
6 Recruiting Pompe Disease Registry
Conditions: Glycogen Storage Disease Type II;   Pompe Disease
Intervention:
7 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
8 Approved for marketing 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
9 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)
10 Terminated
Has Results
A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
Conditions: Pompe Disease (Infantile-Onset);   Glycogen Storage Disease Type II (GSD II);   Glycogenosis 2;   Acid Maltase Deficiency
Intervention: Biological: alglucosidase alfa
11 Completed Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.
Conditions: Pompe Disease;   Glycogen Storage Disease Type II (GSD II);   Acid Maltase Deficiency
Intervention: Drug: GZ402666
12 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:
13 Completed
Has Results
Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa
Conditions: Pompe Disease (Late-Onset);   Glycogen Storage Disease Type II (GSD II);   Glycogenesis 2 Acid Maltase Deficiency
Intervention: Biological: Alglucosidase Alfa
14 Enrolling by invitation An MRI Study on Muscular Diseases -Pompe Disease and Dystrophia Myotonica-
Conditions: Glycogen Storage Disease Type 2;   Dystrophia Myotonica
Intervention:
15 Not yet recruiting Diet and Exercise in Pompe Disease
Conditions: Pompe Disease;   Glycogen Storage Disease Type II;   Lysosomal Storage Diseases
Intervention: Other: Diet and Exercise
16 Recruiting Pompe Pregnancy Sub-Registry
Conditions: Glycogen Storage Disease Type II (GSD-II);   Pompe Disease (Late-onset);   Glycogenesis 2 Acid Maltase Deficiency
Interventions: Biological: alglucosidase alpha;   Other: No Treatment
17 Completed Newborn Screening Assay of Pompe's Disease
Condition: Pompe Disease
Intervention: Other: Pompe disease newborn screening
18 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
19 Recruiting Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
Condition: Pompe Disease
Intervention: Other: Observational
20 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:

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