ZOLGENSMA — evaluated in 4 clinical trials including ongoing and completed studies
aNCT03306277; bNCT02122952; cNCT03421977; dNCT03505099; eAs of March 2019 data cut.
An ongoing, open-label, single-arm clinical trial of symptomatic patients with SMA (N=22)1,2,a
A completed, open-label, single-arm, ascending-dose clinical trial of symptomatic patients with SMA that enrolled a low-dose cohort (n=3) and a high-dose cohort (n=12)3,a
An ongoing, long-term follow-up study of ZOLGENSMA-treated patients from the START clinical trial (N=13)1,4
An ongoing (enrolling), open-label, single-arm clinical trial of presymptomatic patients with SMA5
aAs of March 2019 data cut.
bEvent is defined as death or the need for permanent ventilatory support consisting of ≥16 hours of respiratory assistance per day continuously for ≥14 days.