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.

Keywords

• case report
• elamipretide
• MPAN
• NBIA