CHMP recommends Zolgensma for the treatment of patients with 5q spinal muscular atrophy . - Novartis
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Novartis announced data that reinforce the transformational benefit of Zolgensma (onasemnogene abeparvovec), an essential, one-time treatment and the only gene therapy for spinal muscular atrophy (SMA).
Genentech, a member of the Roche Group announced new interim data from two studies of Evrysdi (risdiplam); JEWELFISH and RAINBOWFISH. Data from JEWELFISH, an ongoing open-label study primarily evaluating the safety of Evrysdi in people aged 1 to 60 years who have been previously treated with another SMA-targeting therapy, including nusinersen and onasemnogene abeparvovec, showed the safety profile of Evrysdi and increase in SMN protein levels are consistent with those observed in other Evrysdi studies.
Genentech, announced new 2-year data from Part 2 of FIREFISH, a Phase II/III global study evaluating Evrysdi (risdiplam) in infants aged 1-7 months at enrollment with symptomatic Type 1 spinal muscular atrophy (SMA).