On December 23, the FDA announced that it has approved SpinrazaTM (nusinersen) to treat spinal muscular atrophy, making it the first-ever FDA-approved therapy for SMA.
We are thrilled to see our community’s efforts culminate in the approval of Spinraza: not only the first-ever approved treatment for this disease, but also one that addresses the underlying genetic cause of SMA. This has been a story of all groups—families, researchers, companies and the FDA—working together as one community to reach this amazing milestone.
The approval from the FDA for all SMA—pediatric and adult—is the broadest possible label, with no restrictions—and this matches our core value at Cure SMA of being one united community for all ages and all types of SMA.
Read more here
FDA Approves Spinraza for SMA
https://www.curesma.org/news/spinraza-approved.html
BY CURE SMA | PUBLISHED ON JANUARY 5, 2017
2018 Updated SMA Drug Pipeline Released
https://www.curesma.org/news/2018-sma-drug-pipeline.html
BY CURE SMA | PUBLISHED ON JANUARY 17, 2018