Mifepristone alone and in combination with scAAV9-SMN1 gene therapy improves disease phenotypes in Smn2B/- spinal muscular atrophy mice
Emma R Sutton, Eve McCallion, Joseph M. Hoolachan, Özge Çetin, Paloma Pacheco-Torres, Saman Rashid, Sihame Bouhmidi, Katie Haynes, Lauren Churchill, Taylor Scaife, Helena Chaytow, Yuting Huang, Stéphanie Duguez, Bernard Laurent Schneider, Thomas Henry Gillingwater, , Mélissa Bowerman
This study shows that mifepristone, a glucocorticoid antagonist, improves SMA disease phenotypes in mice, both alone and when combined with SMN gene therapy.
Scientific Reports · Nov 17, 2025Peer-reviewedPharmacology & Toxicology