Barth Syndrome

Barth Syndrome is the indication for 1 drug in DrugBank. It is covered by 1 curated, structured indication.

Classified under: Congenital, Hereditary, and Neonatal Diseases and Abnormalities
Genetic Variation
Nutritional and Metabolic Diseases
Signs, Symptoms, and General Pathologies
Indicated drugs: Elamipretide

Resolves to

Vocabularies

MeSHM0529363ICD-10E78.71SNOMED CT297231002MedDRA10078537UMLSC0574083

What you can answer from here — as of November 01, 2026

1Indicated drugWith each drug's targets and mechanism

Listed above

1Structured indicationPopulation, line of therapy, route and combination as fields, not prose

5Clinical trialsPhase, status and sponsor resolved per trial

1Drug reporting it as an adverse effectWith incidence where the label states it

Which approved drugs for Barth Syndrome share a target, and which companies have late-stage candidates against it?

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