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

This case reports presents a progressively declining 17-year-old patient with MPAN 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.