Pharmacology & ToxicologyScientific ReportsPeer-reviewed
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…·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.
DOI 10.1038/s41598-025-24050-3