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Randomized dose-escalation trial of elamipretide in adults with primary mitochondrial myopathy

OBJECTIVE: To assess the safety and efficacy of elamipretide, an aromatic-cationic tetrapeptide that readily penetrates cell membranes and transiently localizes to the inner mitochondrial membrane where it associates with cardiolipin, in adults with primary mitochondrial myopathy (PMM). METHODS: A S...

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Detalles Bibliográficos
Autores principales: Karaa, Amel, Haas, Richard, Goldstein, Amy, Vockley, Jerry, Weaver, W. Douglas, Cohen, Bruce H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Lippincott Williams & Wilkins 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5890606/
https://www.ncbi.nlm.nih.gov/pubmed/29500292
http://dx.doi.org/10.1212/WNL.0000000000005255