Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials
71 studies found for:    "glycogen storage disease type IX" OR "Glycogen Storage Disease"
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Rank Status Study
21 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
22 Recruiting Pompe Lactation Sub-Registry
Conditions: Glycogen Storage Disease;   Pompe Disease
Intervention: Biological: alglucosidase alfa
23 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
24 Completed A Prospective, Observational Study in Patients With Late-Onset Pompe Disease
Condition: Glycogen Storage Disease Type II
Intervention:
25 Recruiting Pompe Disease Registry
Conditions: Glycogen Storage Disease Type II;   Pompe Disease
Intervention:
26 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)
27 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
28 Completed 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
29 Recruiting Prospective Follow-up of Patients With Glycogen Storage Disease Type III
Condition: Neuromuscular Disorders
Interventions: Procedure: Different motor function tests;   Procedure: Manual dexterity assessment
30 Unknown  High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe Disease
Condition: Glycogen Storage Disease Type II
Intervention: Other: High Protein and Exercise Therapy along-with Nocturnal Enteral Feeding
31 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
32 Completed
Has Results
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
Conditions: Pompe Disease;   Glycogen Storage Disease Type II
Interventions: Biological: Alglucosidase Alfa;   Drug: Methotrexate;   Drug: Rituximab
33 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
34 Active, not recruiting Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease
Conditions: Adult Polyglucoson Body Disease;   Glycogen Brancher Enzyme Deficiency;   Glycogen Storage Disease Type IV
Interventions: Drug: Triheptanoin;   Dietary Supplement: Vegetable Oil
35 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
36 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:
37 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
38 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
39 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)
40 Recruiting Pharmacokinetics of Alglucosidase Alfa in Patients Aged 8-18 Years of Age
Conditions: Pompe Disease (Late-Onset);   Glycogen Storage Disease Type II (GSD II);   Glycogenesis 2;   Acid Maltase Deficiency
Intervention: Biological: alglucosidase alfa

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