New data back early Zolgensma use in spinal muscular atrophy
Nature Medicine has published final results from the Phase III SPR1NT trial of Zolgensma (onasemnogene abeparvovec) in spinal muscular atrophy (SMA).
The product has been developed by Swiss pharma major Novartis (NOVN: VX).
The results show that, when treated early, certain children with SMA achieved age-appropriate motor milestones, including sitting independently, standing and walking.
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