Genotype-specific effects of elamipretide in patients with primary mitochondrial myopathy: a post hoc analysis of the MMPOWER-3 trial

Karaa, A., Bertini, E., Carelli, V., Cohen, B., Ennes, G. M., Falk, M. J., Goldstein, A., Gorman, G., Haas, R., Hirano, M., Klopstock, T., Koenig, M. K., Kornblum, C., Lamperti, C., Lehman, A., Longo, N., Molnar, M. J., Parikh, S., Phan, H., Pitceathly, R. D. S., … MMPOWER-3 Trial Investigators (2024). 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, 19(1), 431. https://doi.org/10.1186/s13023-024-03421-5

Authors:
Amel Karaa
Enrico Bertini
Valério Carelli
Bruce M. Cohen
Gregory M. Ennes
Marni J. Falk
Amy B. Goldstein
Gráinne S. Gorman
Richard Haas
Michio Hirano
Thomas Klopstock
Mary Kay Koenig
Cornelia Kornblum
Costanza Lamperti
Anna Lehman
Nicola Longo
Mária Judit Molnár
Sumit Parikh
Han Phan
Robert D. S. Pitceathly
Russell P. Saneto
Fernando Scaglia
Serenella Servidei
Mark A. Tarnopolsky
António Toscano
Johan L.K. Van Hove
John Vissing
Jerry Vockley
Jeffrey S. Finman
Anthony Abbruscato
David A. Brown
Alana Sullivan
James A. Shiffer
Michelango Mancuso
Affiliated Authors:
Michio Hirano
Columbia Affiliation:
Subjects:
Genotype (MeSH)
Mitochondrial Myopathies (MeSH)
Oligopeptides (MeSH)
Mitochondrial Dynamics and Reactive Oxygen Species Regulation (OpenAlex Topic)
Metabolic Disorders and Biochemical Genetics (OpenAlex Topic)
ATP Synthase Function and Regulation (OpenAlex Topic)
Author Keywords:
elamipretide
mitochondria
mtdna maintenance
mtdna multiple deletions
pmm
replisome
elink
Publication Type:
Article
Unique ID:
10.1186/s13023-024-03421-5
PMID:
39574155
DOI:
10.1186/s13023-024-03421-5
Journal:
Orphanet Journal of Rare Diseases
Publication Date:
Data Source:
OpenAlex
Source Link:
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Record Created: