62 studies found for:    "Glycogen Storage Disease Type II" OR "Pompe disease"
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"Glycogen Storage Disease Type II" OR "Pompe disease" (62 records)
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1 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
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 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
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 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)
7 Active, not recruiting A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Patients With Infantile-Onset Pompe Disease Who Have Never Been Treated
Conditions: Pompe Disease (Infantile-Onset);   Glycogen Storage Disease Type II (GSD II);   Glycogenosis 2;   Acid Maltase Deficiency
Intervention: Biological: alglucosidase alfa
8 Recruiting 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
9 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:
10 Completed 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
11 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.
Conditions: Pompe Disease Late-Onset;   Glycogen Storage Disease Type II GSD II
Intervention: Biological: Myozyme
12 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
13 Recruiting Pompe Disease Registry
Conditions: Glycogen Storage Disease Type II;   Pompe Disease
Intervention:
14 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
15 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
16 Approved for marketing Alglucosidase Alfa Temporary Access Program
Conditions: Glycogen Storage Disease Type II (GSD-II);   Pompe Disease (Late-Onset);   Acid Maltase Deficiency Disease;   Glycogenosis 2
Intervention: Biological: alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])
17 Recruiting Immune Tolerance Induction Study
Conditions: Pompe Disease;   Glycogen Storage Disease Type II (GSD-II);   Glycogenesis 2 Acid Maltase Deficiency
Intervention: Biological: Myozyme (alglucosidase alfa)
18 Recruiting Growth and Development Study of Myozyme (Alglucosidase Alfa).
Conditions: Pompe Disease;   Glycogen Storage Disease Type II (GSD-II);   Acid Maltase Deficiency Disease
Intervention: Biological: alglucosidase alfa
19 Completed
Has Results
High Dose or High Dose Frequency Study of Alglucosidase Alfa
Conditions: Pompe Disease;   Glycogen Storage Disease Type II (GSD-II);   Glycogenesis 2 Acid Maltase Deficiency
Intervention: Biological: alglucosidase alfa
20 Completed
Has Results
Late-Onset Treatment Study Extension Protocol
Conditions: Pompe Disease (Late-Onset);   Glycogen Storage Disease Type II (GSD-II);   Glycogenesis Type II;   Acid Maltase Deficiency (AMD)
Intervention: Biological: alglucosidase alfa

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