An Intermediate Size Expanded Access Protocol of Elamipretide for Subcutaneous Injection in Patients With Genetically Confirmed Rare Diseases With Known Mitochondrial Dysfunction

Status: Recruiting

Phase of Trial: Clinical Phase Unknown

Latest Information Update: 15 Jul 2024

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Drugs Elamipretide (Primary)
Indications Barth syndrome; Cardiomyopathies; Corneal disorders; Diabetic macular oedema; Dry age-related macular degeneration; Friedreich's ataxia; Heart failure; Leber's hereditary optic atrophy; Mitochondrial disorders; Mitochondrial myopathies; Renal impairment
Focus Expanded access; Therapeutic Use
Sponsors Stealth BioTherapeutics

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