Tofersen treatment in respiratory onset amyotrophic lateral sclerosis and a variant of uncertain significance in SOD1; a case report

European Journal of Human Genetics, Published online: 28 July 2026; doi:10.1038/s41431-026-02208-5

Tofersen treatment in respiratory onset amyotrophic lateral sclerosis and a variant of uncertain significance in SOD1; a case report