Expanded‐Access Use of Elamipretide Improves Quality of Life in Patients With Rare Mitochondrial Disorders Characterized by Ophthalmic Symptoms: A Case Series
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 w...
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| Format: | Article |
| Language: | English |
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Wiley
2024-12-01
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| Series: | Clinical Case Reports |
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| Online Access: | https://doi.org/10.1002/ccr3.9591 |
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| _version_ | 1850105486486011904 |
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| author | Sharique Ansari Mary Kay Koenig |
| author_facet | Sharique Ansari Mary Kay Koenig |
| author_sort | Sharique Ansari |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-941529823dde4b63a1793ac4759af5e8 |
| institution | OA Journals |
| issn | 2050-0904 |
| language | English |
| publishDate | 2024-12-01 |
| publisher | Wiley |
| record_format | Article |
| series | Clinical Case Reports |
| spelling | doaj-art-941529823dde4b63a1793ac4759af5e82025-08-20T02:39:04ZengWileyClinical Case Reports2050-09042024-12-011212n/an/a10.1002/ccr3.9591Expanded‐Access Use of Elamipretide Improves Quality of Life in Patients With Rare Mitochondrial Disorders Characterized by Ophthalmic Symptoms: A Case SeriesSharique Ansari0Mary Kay Koenig1Kane Hall Barry Neurology Bedford Texas USADepartment of Pediatrics, Division of Child & Adolescent Neurology, Center for the Treatment of Pediatric Neurodegenerative Disease University of Texas McGovern Medical School Houston Texas USAABSTRACT 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.https://doi.org/10.1002/ccr3.9591CardiolipinCPEOElamipretidemitochondrial diseaseNARP syndrome |
| spellingShingle | Sharique Ansari 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 Clinical Case Reports Cardiolipin CPEO Elamipretide mitochondrial disease NARP syndrome |
| title | Expanded‐Access Use of Elamipretide Improves Quality of Life in Patients With Rare Mitochondrial Disorders Characterized by Ophthalmic Symptoms: A Case Series |
| title_full | Expanded‐Access Use of Elamipretide Improves Quality of Life in Patients With Rare Mitochondrial Disorders Characterized by Ophthalmic Symptoms: A Case Series |
| title_fullStr | Expanded‐Access Use of Elamipretide Improves Quality of Life in Patients With Rare Mitochondrial Disorders Characterized by Ophthalmic Symptoms: A Case Series |
| title_full_unstemmed | Expanded‐Access Use of Elamipretide Improves Quality of Life in Patients With Rare Mitochondrial Disorders Characterized by Ophthalmic Symptoms: A Case Series |
| title_short | Expanded‐Access Use of Elamipretide Improves Quality of Life in Patients With Rare Mitochondrial Disorders Characterized by Ophthalmic Symptoms: A Case Series |
| title_sort | expanded access use of elamipretide improves quality of life in patients with rare mitochondrial disorders characterized by ophthalmic symptoms a case series |
| topic | Cardiolipin CPEO Elamipretide mitochondrial disease NARP syndrome |
| url | https://doi.org/10.1002/ccr3.9591 |
| work_keys_str_mv | AT shariqueansari expandedaccessuseofelamipretideimprovesqualityoflifeinpatientswithraremitochondrialdisorderscharacterizedbyophthalmicsymptomsacaseseries AT marykaykoenig expandedaccessuseofelamipretideimprovesqualityoflifeinpatientswithraremitochondrialdisorderscharacterizedbyophthalmicsymptomsacaseseries |