Article
Genotype-specific effects of elamipretide in patients with primary mitochondrial myopathy: a post hoc analysis of the MMPOWER-3 trial.
Orphanet journal of rare diseases - 21 Nov 2024
Karaa Amel, Bertini Enrico, Carelli Valerio, Cohen Bruce, Ennes Gregory M, Falk Marni J, Goldstein Amy, Gorman Gráinne, Haas Richard, Hirano Michio, Klopstock Thomas, Koenig Mary Kay, Kornblum Cornelia, Lamperti Costanza, Lehman Anna, Longo Nicola, Molnar Maria Judit, Parikh Sumit, Phan Han, Pitceathly Robert D S, Saneto Russekk, Scaglia Fernando, Servidei Serenella, Tarnopolsky Mark, Toscano Antonio, Van Hove Johan L K, Vissing John, Vockley Jerry, Finman Jeffrey S, Abbruscato Anthony, Brown David A, Sullivan Alana, Shiffer James A, Mancuso Michelango
Abstract excerpt
BACKGROUND: As previously published, the MMPOWER-3 clinical trial did not demonstrate a significant benefit of elamipretide treatment in a genotypically diverse population of adults with primary mitochondrial myopathy (PMM). However, the prespecified subgroup of subjects with disease-causing nuclear DNA (nDNA) pathogenic variants receiving elamipretide experienced an improvement in the six-minute walk test...
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