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Identification of drug modifiers for RYR1-related myopathy using a multi-species discovery pipeline
Ryanodine receptor type I-related myopathies (RYR1-RMs) are a common group of childhood muscle diseases associated with severe disabilities and early mortality for which there are no available treatments. The goal of this study is to identify new therapeutic targets for RYR1-RMs. To accomplish this,...
Autores principales: | Volpatti, Jonathan R, Endo, Yukari, Knox, Jessica, Groom, Linda, Brennan, Stephanie, Noche, Ramil, Zuercher, William J, Roy, Peter, Dirksen, Robert T, Dowling, James J |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
eLife Sciences Publications, Ltd
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7202896/ https://www.ncbi.nlm.nih.gov/pubmed/32223895 http://dx.doi.org/10.7554/eLife.52946 |
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