Expanded-Access Use of Elamipretide Improves Quality of Life in Patients With Rare Mitochondrial Disorders Characterized by Ophthalmic Symptoms: A Case Series.

Ansari S, Koenig MK

Clinical case reports29 Nov 2024Cited by 1 (Europe PMC)

PMID39619320PMCIDPMC11606928DOI10.1002/ccr3.9591PepBankPUB-39619320

Summary

This case series presents the use of elamipretide in two patients with different progressive mitochondrial disorders (chronic progressive external ophthalmoplegia [CPEO] plus and neuropathy, ataxia, and retinitis pigmentosa [NARP] syndrome) characterized by ophthalmic traits. Elamipretide was well tolerated and both patients demonstrated improvement in symptoms while on therapy.

CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study2

2 results from studies that gave the peptide. 2 results from people.

01HumanCase report or seriesSafety outcome
In two patients with progressive mitochondrial disorders, elamipretide was well tolerated.
In plain wordsIn two patients with rare mitochondrial diseases, elamipretide was well tolerated.
Peptide
Elamipretide (SS-31)
Outcome
No significant changeTolerability
Study
n = 2
Outcome group
Adverse events · Whole body
Studied dose
Dose not stated
Source sentences
Summary
Elamipretide was well tolerated
1 context sentence(design, population, supporting numbers)
Summary · context
This case series presents the use of elamipretide in two patients with different progressive mitochondrial disorders (chronic progressive external ophthalmoplegia [CPEO] plus and neuropathy, ataxia, and retinitis pigmentosa [NARP] syndrome) characterized by ophthalmic traits.
Show in the study text Report a problemFND-3961932001
02HumanCase report or seriesEfficacy outcome
In two patients with progressive mitochondrial disorders, both patients showed improvement in symptoms while on elamipretide.
In plain wordsIn two patients with rare mitochondrial diseases, both had better symptoms while taking elamipretide.
Peptide
Elamipretide (SS-31)
Outcome
IncreaseSymptom improvement
Study
n = 2
Outcome group
Mitochondrial function · Whole body
Studied dose
Dose not stated
Source sentences
Summary
both patients demonstrated improvement in symptoms while on therapy.
1 context sentence(design, population, supporting numbers)
Summary · context
This case series presents the use of elamipretide in two patients with different progressive mitochondrial disorders (chronic progressive external ophthalmoplegia [CPEO] plus and neuropathy, ataxia, and retinitis pigmentosa [NARP] syndrome) characterized by ophthalmic traits.
Show in the study text Report a problemFND-3961932002

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Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

Version 2Current
10 Oct 2026, 08:19 UTC
Study results updated
Version 1
9 Oct 2026, 02:47 UTC
First added · source update from Europe PMC

Subjects

Terms as the source lists them.

Publication types

Keywords

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