NeurosciencebioRxivPreprint
Incomplete cerebellar circuit restoration limits functional recovery following SMN therapy in severe spinal muscular atrophy
Sayan Ruwald, Adela Vankova, Frieda Hanschmann…·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.
DOI 10.64898/2026.08.14.744836