Posted by Michael Wonder on 20 Dec 2017
Nusinersen: 'amazing' spinal muscular atrophy drug rejected for Pharmaceutical Benefits Scheme
20 December 2017 - They were hoping for an early Christmas present, but instead the families of children with spinal muscular atrophy are devastated.
A new drug, nusinersen, which has produced remarkable results in some children during a global trial, has been rejected for listing on the PBS.
Spinal muscular atrophy is a muscle-wasting disease, and is the largest genetic cause of death in infant children.
