Biotechnology Swiss Pharma giant Novartis (NOVN: VX) has announced positive new data about Zolgensma (onasemnogene abeparvovec), an essential, one-time treatment and the only gene therapy for spinal muscular atrophy (SMA), from two clinical trials including the completed two-copy cohort of the Phase III SPR1NT clinical trial that demonstrates age-appropriate development when used pre-symptomatically and rapid, clinically meaningful efficacy in symptomatic children, even those with severe SMA at baseline, as well as and the completed Phase III STR1VE-EU trial which demonstrated rapid improvements in motor function following treatment with Zolgensma. 18 June 2021