A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolism

PURPOSE: To evaluate effectiveness of elamipretide in Barth syndrome (BTHS), a genetic condition of defects in TAZ, which causes abnormal cardiolipin on the inner mitochondrial membrane. METHODS: We performed a randomized, double-blind, placebo-controlled crossover trial followed by an open-label ex...

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Detalles Bibliográficos
Autores principales: Reid Thompson, W., Hornby, Brittany, Manuel, Ryan, Bradley, Elena, Laux, Janice, Carr, Jim, Vernon, Hilary J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group US 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7935714/
https://www.ncbi.nlm.nih.gov/pubmed/33077895
http://dx.doi.org/10.1038/s41436-020-01006-8

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7935714/
https://www.ncbi.nlm.nih.gov/pubmed/33077895
http://dx.doi.org/10.1038/s41436-020-01006-8

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