Posted by Michael Wonder on 01 Mar 2021
Nusinersen in spinal muscular atrophy: indication of considerable added benefit in children with an early onset of the disease
1 March 2021 - The treatment delays permanent ventilation of the children and extends life. In addition, the children develop more motor skills.
There is an indication of a considerable additional benefit in children with an early onset of the disease (in the first six months of life) compared to the best possible purely supportive treatment (best supportive care).
