Expanded‐Access Use of Elamipretide Improves Quality of Life in Patients With Rare Mitochondrial Disorders Characterized by Ophthalmic Symptoms: A Case Series
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.
Authors
- Mary Kay Koenig (ORCID: https://orcid.org/0000-0001-5126-8515)
- Sharique Ansari (ORCID: https://orcid.org/0009-0009-0680-6703)
Publication Details
- Journal
- Clinical Case Reports
- Published
- 2024-11-29
- DOI
- https://doi.org/10.1002/ccr3.9591
- Citations
- 1
- Primary Topic
- Mitochondrial Function and Pathology
- Type
- article
- Field-Weighted Citation Impact
- 0.23