Efficacy and safety of risdiplam in adults with 5q-associated spinal muscular atrophy: a nationwide observational cohort study in Austria
EClinicalMedicine. 2025 Sep 26;88:103536. doi: 10.1016/j.eclinm.2025.103536. eCollection 2025 Oct.ABSTRACTBACKGROUND: Spinal muscular atrophy (SMA) is a genetic motor neuron disease marked by the progressive decline of motor function. Risdiplam, an orally administered SMN2 splicing modifier, was app

