Nusinersen Promising for Later-Onset Spinal Muscular Atrophy
Phase 1/2 studies reveal long-term improvement in motor function for children with SMA type II and III
Spinal Muscular Atrophy
First-in-Class Inhibitor Designated Orphan Drug for Spinal Muscular Atrophy
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
Significant improvement in motor function compared with sham procedure among children with SMA
Nusinersen Beneficial in Infantile-Onset Spinal Muscular Atrophy
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
The approval was based on positive data from multiple clinical studies involving >170 patients.
