Results from an open-label Phase 2 clinical trial, NURTURE, evaluating Biogen’s (NASDAQ:BIIB) Spinraza (nusinersen) in 25 presymptomatic patients with spinal muscular atrophy (SMA) showed a long-term treatment benefit. The data are being presented at the Cure SMA Annual SMA Conference in Anaheim, CA and the European Academy of Neurology Annual Congress in Oslo.
After 45.1 months of analysis, all treated patients were alive without the need for permanent ventilation and all were sitting independently. 88% were walking independently.
No new safety signals were reported.
The FDA approved the survival motor neuron-2-directed antisense oligonucleotide for the treatment of SMA in December 2016.
Shares are up 1% premarket on light volume.
Related ticker: Ionis Pharmaceuticals (NASDAQ:IONS)
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