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52 studies found for:    Pompe

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Rank Status Study
21 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
22 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:
23 Not yet recruiting Screening for Early Detection and Prevention of Pompe Disease in Israel Using Tandem Mass Spectrometry
Condition: Pompe Disease
Intervention: Other: Drawing blood spots from Newborns
24 Recruiting Alglucosidase Alfa Pompe Safety Sub-Registry
Condition: Pompe Disease
Intervention: Biological: Alglucosidase alfa
25 Not yet recruiting Detection of Pompe Disease in Adult Patients With Myopathies of Uncertain Origin or With Asymptomatic Hyper-CK-emia
Condition: Pompe Disease
Intervention:
26 Recruiting Biomarker for Pompe Disease
Conditions: Lysosomal Storage Diseases;   Pompe Disease
Intervention:
27 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)
28 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
29 Completed Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602
Condition: Glycogen Storage Disease Type II
Intervention: Biological: Myozyme
30 Active, not recruiting 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
31 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
32 Completed A Prospective, Observational Study in Patients With Late-Onset Pompe Disease
Condition: Glycogen Storage Disease Type II
Intervention:
33 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)
34 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
35 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
36 Recruiting A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease
Condition: Pompe Disease
Intervention: Drug: Alglucosidase alfa
37 Active, not recruiting Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease
Condition: Pompe Disease
Intervention: Biological: BMN 701
38 Terminated 3D Echocardiography Managing Infantile Pompe's Disease
Condition: Congenital Disorders
Intervention:
39 Completed 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
Conditions: Pompe Disease Infantile-Onset;   Glycogen Storage Disease Type II
Intervention: Biological: Myozyme
40 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
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† Indicates status has not been verified in more than two years