Treatment Trials

1 Clinical Trials for Various Conditions

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      Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism
      Description

      This is a compassionate use study to allow patients already taking triheptanoin (C7) through previous studies to continue to receive the supplement. It will also allow triheptanoin supplementation in patients with qualifying disorders if they are failing conventional therapy.

      Conditions
      Very Long-chain acylCoA Dehydrogenase (VLCAD) DeficiencyCarnitine Palmitoyltransferase Deficiencies (CPT1, CPT2)Mitochondrial Trifunctional Protein DeficiencyLong-chain Hydroxyacyl-CoA Dehydrogenase DeficiencyGlycogen Storage DisordersPyruvate Carboxylase Deficiency DiseaseACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY ofBarth Syndrome
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