Key result
A consensus on the underlying cause of FSHD (DUX4 gene de-repression) has enabled targeted therapeutic approaches and increased interest in drug development and clinical trial readiness.
Why the study?
A consensus that FSHD is caused by toxic gain-of-function from DUX4 gene de-repression has enabled targeted treatment development, motivating a review of potential therapeutic approaches and trial readiness.
Design
Review
Authors
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Toolkit equips emerging FSHD sites for trials; supports global infrastructure growth but leaves open effects on recruitment and approvals.
This review outlines the underlying disease mechanism of FSHD (DUX4 gene de-repression) and discusses potential targeted therapeutic approaches and trial readiness.
Wang et al. (2020) conducted a review in Facioscapulohumeral muscular dystrophy (FSHD). Therapeutic approaches targeting DUX4 was evaluated. A consensus on the underlying cause of FSHD (DUX4 gene de-repression) has enabled targeted therapeutic approaches and increased interest in drug development and clinical trial readiness.
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