Dyne Therapeutics’ DYNE-302 normalizes muscular pathology in preclinical FSHD
Oct. 11, 2024
Facioscapulohumeral muscular dystrophy (FSHD) is a severe muscle disorder caused by aberrant DUX4 mRNA expression in skeletal muscle. DUX4 activates downstream target transcriptome, known as D4T, leading to myofiber loss and muscle weakness.