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Myotonic dystrophy type 1 drug development: A pipeline toward the market
Myotonic dystrophy type 1 (DM1) is a multisystemic neuromuscular genetic disease with an estimated prevalence of approximately at least half a million individuals based on its vast ethnic variation. Building upon a well-known physiopathology and several proof-of-concept therapeutic approaches, herei...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier Science Ltd. ;, Distributed by Virgin Mailing and Distribution
2021
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8372527/ https://www.ncbi.nlm.nih.gov/pubmed/33798646 http://dx.doi.org/10.1016/j.drudis.2021.03.024 |