In a 17-year-old patient with membrane protein-associated neurodegeneration, elamipretide treatment was followed by symptomatic improvement in dysarthria.
In plain wordsIn one teenage patient with a progressive brain disease, speech difficulty improved during elamipretide treatment.
- Peptide
- Elamipretide (SS-31)
- Outcome
- IncreaseDysarthria symptom improvement
- Study
n = 1
- Outcome group
- Overall treatment effect · Nervous system and cognition
- Studied dose
- Dose not stated
Source sentences
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.
1 context sentence(design, population, supporting numbers)
Expanded-access use of elamipretide in a patient with membrane protein-associated neurodegeneration.