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

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Trial Profile

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: 13 Jul 2026

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At a glance

  • 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 Mighty Therapeutics

Most Recent Events

  • 08 Jul 2026 According to a Mighty Therapeutics media release, updated data from this trial will be presented at the Barth Syndrome Foundation 2026 International Scientific, Medical, and Family Conference, which is being held on July 19-26, 2026, in Bonita Springs, Florida.
  • 16 Jun 2026 According to Stealth BioTherapeutics media release, Stealth BioTherapeutics is now called Mighty Therapeutics.
  • 04 Jan 2021 New trial record

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