Dyne Therapeutics, Inc., a clinical-stage company specializing in treatments for genetically driven neuromuscular diseases, has unveiled promising preclinical data on their investigational therapy, DYNE-302, for facioscapulohumeral muscular dystrophy (FSHD). In a presentation at the 32nd Annual FSHD Society's International Research Congress, the company highlighted that a single intravenous dose of DYNE-302 restored treadmill running ability in a mouse model of severe FSHD. This was achieved by targeting and reducing the DUX4 mRNA, which is implicated in FSHD pathogenesis. The treatment also corrected muscle damage and inflammation, indicating the potential to reverse existing severe skeletal muscle disease. These findings underscore DYNE-302's promise as a transformative therapy for individuals suffering from this rare, progressive muscle disorder.
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