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Therapeutic Approaches in Facioscapulohumeral Muscular Dystrophy

Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common types of muscular dystrophy affecting roughly 1 in 8,000 individuals. The complex underlying genetics and poor mechanistic understanding has caused a bottleneck in therapeutic development. Until the discovery of DUX4 and its cau...

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Pubblicato in:Trends Mol Med
Autori principali: Cohen, Justin, DeSimone, Alec, Lek, Monkol, Lek, Angela
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2020
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC8048701/
https://ncbi.nlm.nih.gov/pubmed/33092966
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.molmed.2020.09.008
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