Spinal Muscular Atrophy

First-in-Class Inhibitor Designated Orphan Drug for Spinal Muscular Atrophy

By March 29, 2018

By inhibiting the activation of myostatin, SRK-015 may promote a clinically meaningful increase in muscle mass and strength.

Nusinersen Beneficial in Children With Later-Onset Spinal Muscular Atrophy

February 15, 2018

Significant improvement in motor function compared with sham procedure among children with SMA

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.