Barth Syndrome: 8 Clinical Trials

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6

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

Condition(s):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 Disease; ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of; Barth SyndromeLast Updated:December 10, 2021No longer available

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