Expanded-Access Use of Elamipretide in a Patient with Membrane Protein-Associated Neurodegeneration

Authors

Jorge Patino
Anna Haertling Clearman
Lindsey Miller
Mary Kay Koenig

Publication Date

7-1-2024

Journal

Clinical Case Reports

Abstract

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

Comments

PMID: 38919884