Spinal Muscular Atrophy NFZ — 32 facilities in 23 cities

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease causing progressive muscle weakness due to motor neuron loss. It manifests with difficulties in movement, breathing, and swallowing. The NFZ covers genetic testing, physiotherapy, and modern therapies including intrathecal nusinersen, oral risdiplam, and gene therapy with onasemnogene abeparvovec through drug programs.