FSHD / OPMD / EDMD / DMI

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Autosomal dominant Facioscapulohumeral muscular dystrophy (FSHD) is among the most prevalent muscular dystrophies, estimated to affect 1 in 8,333-20,000 individuals worldwide. FSHD is linked to aberrant expression of the DUX4 gene, which encodes a myotoxic transcription factor. Since DUX4 is extremely toxic, animal model development has been difficult, but progress has been made, revealing that tight regulation of DUX4 expression is critical for creating a viable model that develops myopathic features that are useful as therapeutic outcome measures.

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