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Current Therapeutic Approaches in FSHD

Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common muscular dystrophies. Over the last decade, a consensus was reached regarding the underlying cause of FSHD allowing—for the first time—a targeted approach to treatment. FSHD is the result of a toxic gain-of-function from de-repr...

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Detalles Bibliográficos
Autores principales: Wang, Leo H., Tawil, Rabi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8203219/
https://www.ncbi.nlm.nih.gov/pubmed/33579868
http://dx.doi.org/10.3233/JND-200554