For which patients is treatment with Tofersen available?

Around 2% of all people with ALS have genetic mutations in the superoxide dismutase 1 (SOD1) gene. The mutations in the SOD1 gene are responsible for ALS in these patients. The harmful effects of the mutations in the SOD1 gene can be reduced by the genetic drug Tofersen (trade name Qalsody). The drug has been available in Germany in a hardship program since the beginning of 2022 and has been in use at Charité and other specialized ALS centers since then. Experience to date shows that it is very well tolerated and effective for the majority of those affected. The effect can be seen in a slowing or halting of ALS. Individual patients have shown an improvement in motor functions.