Use of Elamipretide in patients assigned treatment in the compassionate use program: Case series in pediatric patients with rare orphan diseases

Several mitochondrial diseases are caused by pathogenic variants that impair membrane phospholipid remodeling, with no FDA‐approved therapies. Elamipretide targets the inner mitochondrial membrane where it binds to cardiolipin, resulting in improved membrane stability, cellular respiration, and ATP...

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Detalles Bibliográficos
Autores principales: Koenig, Mary Kay, Russo, Sam Nick, McBride, Kim L., Bjornsson, Hans Tomas, Gunnarsdottir, Brynja Bjork, Goldstein, Amy, Falk, Scott A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2022
Materias:
Research Reports
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9830009/
https://www.ncbi.nlm.nih.gov/pubmed/36636586
http://dx.doi.org/10.1002/jmd2.12335

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9830009/
https://www.ncbi.nlm.nih.gov/pubmed/36636586
http://dx.doi.org/10.1002/jmd2.12335

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