Spinraza

Spinraza is a medicine used to treat 5q spinal muscular atrophy (SMA), a genetic disease that causes weakness and wasting of the muscles including the lung muscles. The disease is linked to a defect on chromosome 5q and symptoms usually start shortly after birth. SMA is rare, and Spinraza was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 2 April 2012. Further information on the orphan designation can be found on the [EMA website](https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-976). Spinraza contains the active substance nusinersen.