64 studies found for:    "glycogen storage disease type IX" OR "Glycogen Storage Disease"
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Rank Status Study
1 Recruiting Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard Versus Modified Uncooked Cornstarch
Conditions: Glycogen Storage Disease Type IA;   Glycogen Storage Disease Type IB;   Glycogen Storage Disease Type III;   Glycogen Storage Disease Type 0
Intervention:
2 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
3 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
4 Completed Study of Glycogen Storage Disease and Associated Disorders
Condition: Glycogen Storage Disease
Intervention:
5 Recruiting Study of the Relationship Between Glycogen Storage Disease Type Ia and Inflammatory Bowel Disease
Conditions: Inflammatory Bowel Disease;   Glycogen Storage Disease Type Ia
Intervention: Other: Glycogen Storage Disease type Ia patients
6 Recruiting Study of Glycogen Storage Disease Expression in Carriers
Condition: Glycogen Storage Disease
Intervention: Genetic: Glycogen Storage Disease markers
7 Completed Genetic and Family Studies of Inherited Muscle Diseases
Conditions: Dermatomyositis;   Glycogen Storage Disease Type II;   Glycogen Storage Disease Type VII;   Myositis;   Polymyositis
Intervention:
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 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
10 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
11 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
12 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
13 Not yet recruiting Overnight Feeding Study in Glycogen Storage Disease Type 1
Condition: Glycogen Storage Disease Type 1 (GSD 1)
Intervention: Other: overnight nutrition regime
14 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])
15 Available Compassionate Use of Triheptanoin (C7) for Fatty Acid Oxidation Disorders and Glycogen Storage Disease
Conditions: Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency;   Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2);   Mitochondrial Trifunctional Protein Deficiency;   Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency;   Glycogen Storage Disorders;   Pyruvate Carboxylase Deficiency, Type B;   Acyl-coA Dehydrogenase, Type 9
Intervention: Drug: triheptanoin
16 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)
17 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
18 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
19 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
20 Recruiting Pompe Lactation Sub-Registry
Conditions: Glycogen Storage Disease;   Pompe Disease
Intervention: Biological: alglucosidase alfa

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