Identification of compounds that repress DUX4 expression in facioscapulohumeral muscular dystrophy
Ning Chang, Hannah P. Moore, Charis L. Himeda, Terrence E. O’Brien, William Thomas, Baback Roshanravan, Takako I. Jones, Peter L. Jones
This study identifies a compound, C06, that effectively represses DUX4 expression in FSHD myocytes using AI-based screening and validation.
Scientific Reports · Jun 16, 2026Peer-reviewedPharmacology & Toxicology