Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials
3 studies found for:    "glycogen storage disease type VII" [DISEASE] OR "Tarui disease" [DISEASE] OR "phosphofructokinase deficiency" [DISEASE] OR NCT01461304 [ID-NUMBER] OR NCT02057731 [ID-NUMBER]
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Rank Status Study
1 Completed Genetic and Family Studies of Inherited Muscle Diseases
Conditions: Dermatomyositis;   Glycogen Storage Disease Type II;   Glycogen Storage Disease Type VII;   Myositis;   Polymyositis
Intervention:
2 Recruiting Study of Glycogen Storage Disease Expression in Carriers
Condition: Glycogen Storage Disease
Intervention: Genetic: Glycogen Storage Disease markers
3 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

Indicates status has not been verified in more than two years