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 Spinraza^®, 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 Evrysdi^®, a treatment developed by Genentech/Roche, to treat SMA in adults and children 2 months and older, and updated in 2022 to include all ages.

Cure SMA provided the very first research funding for these drug development programs.

Learn more about these life-saving treatments and others in development.

For additional guidance on SMA treatment for newly diagnosed families, click here.

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