Data edition of
Residue An evidence-graded reference for peptide science

Elamipretide research

What PubMed holds on Elamipretide, sorted by study design: the papers this site relies on, every clinical-trial publication PubMed tags, and the newest additions.

PubMed records
470
Trial publications
2119 randomized
Reviews
97
Human-subject tagged
201
Animal-only
170
  • Clinical-trial publications 21
  • Reviews 97
  • Animal-only studies 170
  • Other (in vitro, chemistry, commentary, unindexed) 182
Figure 1. Composition of the Elamipretide literature by PubMed publication type and MeSH tags. Categories come from separate PubMed filters and can overlap; “animal-only” means tagged Animals and not Humans. Query: (elamipretide[tiab] OR SS-31[tiab] OR MTP-131[tiab] OR bendavia[tiab]).
03672 1993: 1 records 1993 1994: 0 records 1995: 0 records 1995 1996: 0 records 1997: 3 records 1998: 0 records 1999: 0 records 2000: 1 records 2000 2001: 0 records 2002: 0 records 2003: 1 records 2004: 1 records 2005: 1 records 2005 2006: 2 records 2007: 2 records 2008: 2 records 2009: 3 records 2010: 2 records 2010 2011: 8 records 2012: 5 records 2013: 10 records 2014: 20 records 2015: 18 records 2015 2016: 18 records 2017: 26 records 2018: 31 records 2019: 30 records 2020: 31 records 2020 2021: 40 records 2022: 40 records 2023: 32 records 2024: 59 records 2025: 72 records 2025 2026: 72 records (year to date) 2026
Figure 2. PubMed records per publication year matching (elamipretide[tiab] OR SS-31[tiab] OR MTP-131[tiab] OR bendavia[tiab]) — 470 in total; peak 72 in 2025. The 2026 bar is year-to-date (hatched). Volume of literature is not evidence of efficacy. Run this query on PubMed.

Studies this site relies on

Ordered by strength of design. Conclusions are quoted verbatim from each abstract as indexed by PubMed; read the full paper before relying on any of them.

  1. Phase 3 trial Neurology · 2023 PMID 37268435

    Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial

    Karaa A, Bertini E, Carelli V, et al.

    Subcutaneous elamipretide treatment did not improve outcomes in the 6MWT and PMMSA TFS in patients with PMM. However, this phase-3 study demonstrated that subcutaneous elamipretide is well-tolerated.

  2. Phase 3 trial Genet Med · 2021 PMID 33077895

    A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolism

    Reid Thompson W, Hornby B, Manuel R, et al.

    In this interventional clinical trial in BTHS, daily administration of elamipretide led to improvement in BTHS symptoms.

  3. Randomized trial Genet Med · 2024 PMID 38602181

    Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER

    Thompson WR, Manuel R, Abbruscato A, et al.

    Elamipretide was associated with sustained long-term tolerability and efficacy, with improvements in functional assessments and cardiac function in BTHS.

  4. Randomized trial Circ Heart Fail · 2017 PMID 29217757

    Novel Mitochondria-Targeting Peptide in Heart Failure Treatment: A Randomized, Placebo-Controlled Trial of Elamipretide

    Daubert MA, Yow E, Dunn G, et al.

    This is the first study to evaluate elamipretide in heart failure with reduced ejection fraction and demonstrates that a single infusion of elamipretide is safe and well tolerated. High-dose elamipretide resulted in favorable changes in left ventricular volumes that correlated with peak plasma concentrations, supporting a temporal …

  5. Review Drugs · 2026 PMID 41335372

    Elamipretide: First Approval

    Shirley M

    Elamipretide is also under phase III clinical development for use in the treatment of dry age-related macular degeneration and mitochondrial myopathies. This article summarizes the milestones in the development of elamipretide leading to this first approval for Barth syndrome.

Clinical-trial publications in PubMed

21 records carry PubMed’s clinical-trial publication type. Tagging is done by NLM indexers and occasionally includes secondary analyses.

Clinical-trial publications on Elamipretide
YearTitleJournalDesign
2025Initial Psychometric Evaluation of the Barth Syndrome Symptom Assessment (BTHS-SA) for Adolescents and Adults in a Phase 2 Clinical StudyOrphanet J Rare DisPhase 2 trial
2024Genotype-specific effects of elamipretide in patients with primary mitochondrial myopathy: a post hoc analysis of the MMPOWER-3 trialOrphanet J Rare DisPhase 3 trial
2024Effect of Aficamten on Health Status Outcomes in Obstructive Hypertrophic Cardiomyopathy: Results From SEQUOIA-HCMJ Am Coll CardiolPhase 3 trial
2024Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWERGenet MedRandomized trial
2024Elamipretide Topical Ophthalmic Solution for the Treatment of Subjects with Leber Hereditary Optic Neuropathy: A Randomized TrialOphthalmologyPhase 2 trial
2023Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical TrialNeurologyPhase 3 trial
2023Identifying responders to elamipretide in Barth syndrome: Hierarchical clustering for time series dataOrphanet J Rare DisRandomized trial
2022Natural history comparison study to assess the efficacy of elamipretide in patients with Barth syndromeOrphanet J Rare DisPhase 3 trial
2021In vivo mitochondrial ATP production is improved in older adult skeletal muscle after a single dose of elamipretide in a randomized trialPLoS OneRandomized trial
2021A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolismGenet MedPhase 3 trial
2020A randomized crossover trial of elamipretide in adults with primary mitochondrial myopathyJ Cachexia Sarcopenia MuscleRandomized trial
2020Effects of Elamipretide on Left Ventricular Function in Patients With Heart Failure With Reduced Ejection Fraction: The PROGRESS-HF Phase 2 TrialJ Card FailPhase 2 trial
2019The mitochondria-targeting peptide elamipretide diminishes circulating HtrA2 in ST-segment elevation myocardial infarctionEur Heart J Acute Cardiovasc CarePhase 2 trial
2018Randomized dose-escalation trial of elamipretide in adults with primary mitochondrial myopathyNeurologyPhase 2 trial
2017Novel Mitochondria-Targeting Peptide in Heart Failure Treatment: A Randomized, Placebo-Controlled Trial of ElamipretideCirc Heart FailRandomized trial
2017Phase 2a Clinical Trial of Mitochondrial Protection (Elamipretide) During Stent Revascularization in Patients With Atherosclerotic Renal Artery StenosisCirc Cardiovasc IntervPhase 2 trial
2016Relation of Left Ventricular Mass and Infarct Size in Anterior Wall ST-Segment Elevation Acute Myocardial Infarction (from the EMBRACE STEMI Clinical Trial)Am J CardiolPhase 2 trial
2016Effects of Single Vs. Multiple Sets during 10 Weeks of Water-based Resistance Training on Neuromuscular Adaptations in Young WomenInt J Sports MedRandomized trial
2016EMBRACE STEMI study: a Phase 2a trial to evaluate the safety, tolerability, and efficacy of intravenous MTP-131 on reperfusion injury in patients undergoing primary percutaneous coronary interventionEur Heart JPhase 2 trial
2012Comparison of the pharmacokinetics of a new 30 mg modified-release tablet formulation of metoclopramide for once-a-day administration versus 10 mg immediate-release tablets: a single and multiple-dose, randomized, open-label, parallel study in healthy male subjectsEur J Drug Metab PharmacokinetRandomized trial
1977Antibodies to pancreatic duct cells in Sjögren's syndrome and rheumatoid arthritisGutClinical trial

Newest records

The most recent PubMed additions for this query at the data-edition date, regardless of design.

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