Spinal Muscular Atrophy

Nusinersen Beneficial in Infantile-Onset Spinal Muscular Atrophy

November 02, 2017

In additiona, a gene replacement therapy was found to be beneficial in spinal muscular atrophy type 1 (SMA1).

First Treatment for Spinal Muscular Atrophy Gets FDA Approval

By December 27, 2016

The approval was based on positive data from multiple clinical studies involving >170 patients.