Novartis presents data from Phase III study of Zolgensma (onasemnogene abeparvovec) in patients with presymptomatic spinal muscular atrophy (SMA)

Novartis reports that in the Phase III SPRINT study, Zolgensma lead to age-appropriate milestones, with 100% of the 14 children in the two-copy cohort treated presymptomatically able to sit independently for longer than 30 seconds.

SPS commentary:

Zolgensma is licensed in the UK for patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1, or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.

Biospace Inc.