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17 November 2025
  • Mifepristone alone and in combination with scAAV9- SMN1 gene therapy improves disease phenotypes in Smn 2B/- spinal muscular atrophy mice. (2025) 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, Yu-Ting Huang, Stephanie Duguez, Bernard L. Schneider, Thomas H. Gillingwater, Maria Dimitriadi and Melissa Bowerman