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

oleh: Jorge Patino, Anna Haertling Clearman, Lindsey Miller, Mary Kay Koenig

Format: Article
Diterbitkan: Wiley 2024-07-01

Deskripsi

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.