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Barth syndrome cardiomyopathy: targeting the mitochondria with elamipretide
Barth syndrome (BTHS) is a rare, X-linked recessive, infantile-onset debilitating disorder characterized by early-onset cardiomyopathy, skeletal muscle myopathy, growth delay, and neutropenia, with a worldwide incidence of 1/300,000–400,000 live births. The high mortality rate throughout infancy in...
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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Springer US
2020
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7895793/ https://www.ncbi.nlm.nih.gov/pubmed/33001359 http://dx.doi.org/10.1007/s10741-020-10031-3 |