Today, families with spinal muscular atrophy (SMA) have new hope. A life-saving therapy — 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. This is the first-ever approved therapy for this devastating disease.
Cure SMA provided the very first research funding for this drug development program beginning in 2003.
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