1RCT2020
Karaa A, Haas R, Goldstein A, Vockley J, Cohen BH · J Cachexia Sarcopenia Muscle (2020)
No plain-language summary recorded for this citation — open the paper to read its abstract.
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22 peer-reviewed studies
What the evidence says
Most Elamipretide studies are mechanism or observational rather than RCTs that measure a clinical effect — keep findings provisional.
Most evidence is from high-quality meta-analyses and randomised trials published 2016–2026 with a typical study size of 19 participants.
Based on 22 studies · 1 meta-analysis · 14 RCTs · 58 total participants
Confidence
Moderate confidenceBy outcome
15 studies in the last 5 years · Latest meta-analysis: 2022
Karaa A, Haas R, Goldstein A, Vockley J, Cohen BH · J Cachexia Sarcopenia Muscle (2020)
No plain-language summary recorded for this citation — open the paper to read its abstract.
Saad A, Herrmann SMS, Eirin A, Ferguson CM, Glockner JF, Bjarnason H · Circ Cardiovasc Interv (2017)
No plain-language summary recorded for this citation — open the paper to read its abstract.
Roshanravan B, Liu SZ, Ali AS, Shankland EG, Goss C, Amory JK · PLoS One (2021)
No plain-language summary recorded for this citation — open the paper to read its abstract.
Van den Eynde J, Chinni B, Vernon H, Thompson WR, Hornby B, Kutty S · Orphanet J Rare Dis (2023)
No plain-language summary recorded for this citation — open the paper to read its abstract.
Reid Thompson W, Hornby B, Manuel R, Bradley E, Laux J, Carr J · Genet Med (2021)
No plain-language summary recorded for this citation — open the paper to read its abstract.
Daubert MA, Yow E, Dunn G, Marchev S, Barnhart H, Douglas PS · Circ Heart Fail (2017)
No plain-language summary recorded for this citation — open the paper to read its abstract.
Thompson WR, Manuel R, Abbruscato A, Carr J, Campbell J, Hornby B · Genet Med (2024)
No plain-language summary recorded for this citation — open the paper to read its abstract.
Karaa A, Bertini E, Carelli V, Cohen BH, Enns GM, Falk MJ · Neurology (2023)
No plain-language summary recorded for this citation — open the paper to read its abstract.
Karaa A, Bertini E, Carelli V, Cohen B, Ennes GM, Falk MJ · Orphanet J Rare Dis (2024)
No plain-language summary recorded for this citation — open the paper to read its abstract.
Hortmann M, Robinson S, Mohr M, Mauler M, Stallmann D, Reinöhl J · Eur Heart J Acute Cardiovasc Care (2019)
No plain-language summary recorded for this citation — open the paper to read its abstract.
Karaa A, Haas R, Goldstein A, Vockley J, Weaver WD, Cohen BH · Neurology (2018)
No plain-language summary recorded for this citation — open the paper to read its abstract.
Butler J, Khan MS, Anker SD, Fonarow GC, Kim RJ, Nodari S · J Card Fail (2020)
No plain-language summary recorded for this citation — open the paper to read its abstract.
In plain English: Furthermore, we offer a comprehensive outlook on the expansive prospects of SS-31's future development and application.
Du X, Zeng Q, Luo Y, He L, Zhao Y, Li N, Han C, Zhang G, Liu W. · Mitochondrion (2024)
In plain English: A comprehensive understanding of mitochondrial redox processes during gestational diabetes offers unique potential for improving pregnancy outcomes and may contribute to reducing the intergenerational inheritance of metabolic diseases.
Burzynska-Pedziwiatr I, Wozniak LA, Bukowiecka-Matusiak M. · Free radical biology & medicine (2026)
In plain English: Administration of MTP-131 was not associated with a significant reduction in the primary endpoint, infarct size by CK-MB AUC over 72 h, nor with improvement in prespecified MRI, angiographic, electrocardiographic, or clinical outcomes.
Gibson CM, Giugliano RP, Kloner RA, Bode C, Tendera M, Jánosi A, et al. · Eur Heart J (2016)
Gwaltney C, Shields A, Love E, Ollis S, Stokes J, Mazar I, Arenson E, Aiudi A, Wirth RJ, Houts C. · Orphanet journal of rare diseases (2025)
In plain English: Herein we evaluate the emerging role of PLSCR3 as a potentially druggable mitochondrial target, supported by recent genetic, biochemical, and in vivo evidence, and discuss translational strategies that may bridge the gap between experimental promise and clinical application.
Patel PS, Pabla NS, Bajwa A. · Seminars in nephrology (2026)
In plain English: While short-term studies indicate that mitoAOXs are generally well tolerated, there is currently limited evidence to support the use of mitoAOXs in the management of glycaemic control and cardiovascular health.
Mason SA, Wadley GD, Keske MA, Parker L. · Diabetes Obes Metab (2022)
In plain English: [This] mitochondria-targeting tetrapeptide selectively binds cardiolipin (CL), a lipid found in the inner mitochondrial membrane, thus stabilizing mitochondrial cristae structure, reducing oxidative stress, and enhancing adenosine [triphosphate production].
Tung C, Varzideh F, Farroni E, Mone P, Kansakar U, Jankauskas SS, et al. · Int J Mol Sci (2025)
In plain English: For the 6-minute walk test, the least-squares mean difference between elamipretide-treated patients and natural-history controls was 79.7 m (P = 0.0004) at week 64.
Hornby B, Thompson WR, Almuqbil M, Manuel R, Abbruscato A, Carr J, et al. · Orphanet J Rare Dis (2022)