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Article

FSHD Therapeutic Strategies: What Will It Take to Get to Clinic?

25 May 2022

Abstract excerpt

Facioscapulohumeral muscular dystrophy (FSHD) is arguably one of the most challenging genetic diseases to understand and treat. The disease is caused by epigenetic dysregulation of a macrosatellite repeat, either by contraction of the repeat or by mutations in silencing proteins. Both cases lead to chromatin relaxation and, in the context of a permissive allele, pathogenic misexpression of DUX4 in skeletal...

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PMID: 35743650
DOI: 10.3390/jpm12060865

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