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

Authors

• Sharique Ansari
• Mary Kay Koenig

Language

English

Publication Date

12-1-2024

Journal

Clinical Case Reports

DOI

10.1002/ccr3.9591

PMID

39619320

PMCID

PMC11606928

PubMedCentral® Posted Date

11-29-2024

PubMedCentral® Full Text Version

Post-print

Abstract

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.

Keywords

Cardiolipin, CPEO, Elamipretide, mitochondrial disease, NARP syndrome

Published Open-Access

yes

Recommended Citation

Sharique Ansari and Mary Kay Koenig, "Expanded-Access Use of Elamipretide Improves Quality of Life in Patients With Rare Mitochondrial Disorders Characterized by Ophthalmic Symptoms: A Case Series" (2024). Faculty, Staff and Student Publications. 4201.
https://digitalcommons.library.tmc.edu/uthmed_docs/4201