NEW THERAPEUTIC APPROACHES

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Autosomal dominant Facioscapulohumeral muscular dystrophy (FSHD) is among the most prevalent muscular dystrophies, affecting 1 in 8,333 to 1 in 20,000 individuals. The clinical features of FSHD were first described in the 1800’s, and the disease was formally classified as a major form of muscular dystrophy in 1954, but the pathogenic mechanisms underlying FSHD have only begun clarifying during the last decade.  This lag in understanding can be largely attributed to the complicated nature of FSHD pathogenesis.  Importantly, FSHD has now been linked to de-repression of the toxic DUX4 gene in muscle.  The emergence of DUX4 represented a momentum shift in the FSHD field as it facilitated the development of cell and animal models and provided a target for rational therapy design centered on inhibiting DUX4 expression or activity.  In this session, several newly emerging FSHD-targeted therapeutic strategies will be discussed, as well as possible paths forward toward clinical development.

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