Expanded-access use of elamipretide in a patient with membrane protein-associated neurodegeneration.

Autor: Patino J; Department of Neurology University of Texas McGovern Medical School Houston Texas USA., Clearman AH; Division of Child and Adolescent Neurology, Department of Pediatrics, Center for the Treatment of Pediatric Neurodegenerative Disease University of Texas McGovern Medical School Houston Texas USA., Miller L; Division of Child and Adolescent Neurology, Department of Pediatrics, Center for the Treatment of Pediatric Neurodegenerative Disease University of Texas McGovern Medical School Houston Texas USA., Koenig MK; Division of Child and Adolescent Neurology, Department of Pediatrics, Center for the Treatment of Pediatric Neurodegenerative Disease University of Texas McGovern Medical School Houston Texas USA.
Jazyk: angličtina
Zdroj: Clinical case reports [Clin Case Rep] 2024 Jun 24; Vol. 12 (7), pp. e9116. Date of Electronic Publication: 2024 Jun 24 (Print Publication: 2024).
DOI: 10.1002/ccr3.9116
Abstrakt: This case report presents a progressively declining 17-year-old patient with membrane protein-associated neurodegeneration who demonstrated symptomatic improvements in her dysarthria, dysphagia, and gait, and objective improvements in her 6-minute walk test and 5 times sit-to-stand test during elamipretide treatment.
Competing Interests: The authors have no conflicts of interest to declare.
(© 2024 The Author(s). Clinical Case Reports published by John Wiley & Sons Ltd.)
Databáze: MEDLINE
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