archived version — not current
This is a permanent, read-only archive of this profile's public content as it existed on 2026-09-27. It does not reflect the current, live profile. View the current profile.
Elamipretide
Snapshot captured 2026-09-27 — Evidence added or removed
Overview
Mitochondria-targeting aromatic-cationic tetrapeptide that interacts with cardiolipin; evidence is separated by disease and endpoint.
Evidence quality
Source identities are PubMed-resolved and provisionally graded by study design. Full-text findings, bias, applicability, contradictions, and regulatory interpretation remain pending.
Safety concerns
US accelerated approval for a limited Barth-syndrome population must not be generalized to other proposed uses.
Citations as of this version
- Beyond the injection: delivery systems reshaping retinal disease management.
- Is an emerging pharmacotherapeutic era for rare mitochondrial diseases here? — Cell metabolism (2026)
- Safety and Efficacy of Approved and Unapproved Peptide Therapies for Musculoskeletal Injuries and Athletic Performance. — Sports medicine (Auckland, N.Z.) (2026)
- RePOWER: An International, Prospective, Non-Interventional Registry of Patients With Primary Mitochondrial Myopathy. — Clinical genetics (2026)
- Elamipretide: First Approval. — Drugs (2026)
- Synergistic mitochondrial homeostasis regulation and cholinergic circuits reconstruction via a one-step synthesized multifunctional hydrogel facilitates spinal cord injury repair. — Bioactive materials (2026)
- Mitochondrial Dysfunction: The Silent Catalyst of Kidney Disease Progression. — Cells (2025)
- Elamipretide: A Review of Its Structure, Mechanism of Action, and Therapeutic Potential. — International journal of molecular sciences (2025)
- SS-31@Fer-1 Alleviates ferroptosis in hypoxia/reoxygenation cardiomyocytes via mitochondrial targeting. — Biomedicine & pharmacotherapy = Biomedecine & pharmacotherapie (2025)
- Targeting ANT1 to regulate PINK1/Parkin-mediated mitophagy is an effective treatment of trauma-induced tendon heterotopic ossification. — Journal of orthopaedic translation (2025)
- SS-31 Targets NOS2 to Enhance Osteogenic Differentiation in Aged BMSCs by Restoring Mitochondrial Function. — Organogenesis (2025)
- Examination of mitochondria- and inflammasome-mediated mechanisms of clozapine-induced myocarditis using patient-derived iPSC cardiomyocytes. — Molecular psychiatry (2025)
- Telomere length in offspring is determined by mitochondrial-nuclear communication at fertilization. — Nature communications (2025)
- Genome-Wide CRISPR Screen Identifies Phospholipid Scramblase 3 as the Biological Target of Mitoprotective Drug SS-31. — Journal of the American Society of Nephrology : JASN (2024)
- Genotype-specific effects of elamipretide in patients with primary mitochondrial myopathy: a post hoc analysis of the MMPOWER-3 trial. — Orphanet journal of rare diseases (2024)
- ALCAT1-mediated abnormal cardiolipin remodelling promotes mitochondrial injury in podocytes in diabetic kidney disease. — Cell communication and signaling : CCS (2024)
- Effect of Aficamten on Health Status Outcomes in Obstructive Hypertrophic Cardiomyopathy: Results From SEQUOIA-HCM. — Journal of the American College of Cardiology (2024)
- Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial. — Neurology (2023)
- Quality of life in Barth syndrome. — Therapeutic advances in rare disease (2022)
- Elamipretide for Barth syndrome cardiomyopathy: gradual rebuilding of a failed power grid. — Heart failure reviews (2022)
- A ROS-Responsive Liposomal Composite Hydrogel Integrating Improved Mitochondrial Function and Pro-Angiogenesis for Efficient Treatment of Myocardial Infarction. — Advanced healthcare materials (2022)
- Targeting mitochondrial dysfunction with elamipretide. — Heart failure reviews (2022)
- Potential Therapeutic Candidates for Age-Related Macular Degeneration (AMD). — Cells (2021)
- Mitochondrial protein interaction landscape of SS-31. — Proceedings of the National Academy of Sciences of the United States of America (2020)
- Elamipretide (SS-31) improves mitochondrial dysfunction, synaptic and memory impairment induced by lipopolysaccharide in mice. — Journal of neuroinflammation (2019)
- Novel cell-penetrating peptide targeting mitochondria. — FASEB journal : official publication of the Federation of American Societies for Experimental Biology (2015)
Regulatory status as of this version
- United States: approved — Improve muscle strength in adults and pediatric patients with Barth syndrome weighing at least 30 kg.
- European Union: not_approved
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