Clinical Case Reports (Jul 2024)
Expanded‐access use of elamipretide in a patient with membrane protein‐associated neurodegeneration
Abstract
Key Clinical Message 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.
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