Incomplete cerebellar circuit restoration limits functional recovery following SMN therapy in severe spinal muscular atrophy
Sayan Ruwald, Adela Vankova, Frieda Hanschmann, Christian Menedo, Sandra Wittig, Marie L Stephan, Vanessa Dreilich, Severine Ruetze, Amy K. Smith, Leonie Sowoidnich, Christian Geis, Stefan Hallermann, Charlotte J. Sumner, Livio Pellizzoni, Beatriz Blanco-Redondo, , Christian M. Simon
The paper identifies cerebellar pathology as a conserved yet distinct feature in severe SMA, showing divergent therapeutic outcomes with SMN-restoring therapies.
bioRxiv · Aug 19, 2026 · PreprintNeuroscience