Today, families with spinal muscular atrophy (SMA) have new hope. Life-saving therapies — and a robust pipeline of other promising treatments on the horizon — are now available for children diagnosed with SMA. Early diagnosis and early treatment are key.
See why early diagnosis and access to treatment are essential SMArt Moves for these families and children with SMA. And if you’re a healthcare professional, learn what you can do as you evaluate children with SMA:
Until recently, there were no treatment options available for children diagnosed with spinal muscular atrophy (SMA), a serious and life-threatening neuromuscular disease. In December 2016, the FDA announced the approval of SpinrazaTM, a treatment developed by Biogen and Ionis, to treat all types of SMA. Then, in May 2019, the FDA announced the approval of Zolgensma®, a treatment developed by Novartis Gene Therapies, to also treat all types of SMA in patients who are under two years of age. Finally, in August 2020, the FDA announced the approval of EvrysdiTM, a treatment developed by Genentech, a member of the Roche Group, to treat SMA in adults and children 2 months and older.
Cure SMA provided the very first research funding for these drug development programs.
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