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-Reference-Cited by-同舟云学术

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

Published:2021-03 Issue:3 Volume:23 Page:471-478
ISSN:1098-3600
Container-title:Genetics in Medicine
language:en
Short-container-title:Genetics in Medicine

Author:

Reid Thompson W.,Hornby Brittany,Manuel Ryan,Bradley Elena,Laux Janice,Carr Jim,Vernon Hilary J.^ORCID

Publisher

Elsevier BV

Subject

Genetics (clinical)

Link

https://www.nature.com/articles/s41436-020-01006-8.pdf

Reference33 articles.

1. Ferreira C, Pierre G, et al. Barth syndrome. 2014 Oct [Updated 2020 Jul 9]. In: Adam MP, Ardinger HH, Pagon RA, et al., editors. GeneReviews. Seattle, WA: University of Washington; 1993-2020.

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