START (long-term follow-up): ZOLGENSMA demonstrates durability of effect nearly 4 years after treatment1

A total of 10 out of 12 high-dose patients voluntarily enrolled in an ongoing, observational, long-term follow-up of the START trial.1

As of March 8, 2019, the mean time since treatment was 3.7 years (range 3.3-4.3) and the mean age at last follow-up was 3.9 years (range 3.4-4.8)1

All 10 of the patients from the high-dose cohort who enrolled in the long-term follow-up were alive and free of permanent ventilation as of the March 2019 data cut1,a

aEvent is defined as death or the need for permanent ventilatory support consisting of ≥16 hours of respiratory assistance per day continuously for ≥14 days.
bNatural history: The Pediatric Neuromuscular Clinical Research (PNCR) Network study population (n=23), with bi-allelic deletion of SMN1 gene, 2 copies of SMN2, and onset of SMA symptoms at age ≤6 months, was used as a matched control cohort for START and STR1VE studies.2
cA 15-year, voluntary follow-up of patients in the START trial who received a high-dose of ZOLGENSMA (n=10).1

START (long-term follow-up): Motor milestone achievements are sustained post treatment1

There has been no loss of milestones achieved during START among 10 patients from the high-dose cohort who enrolled in the long-term follow-up (LTFU)1

aHigh-dose cohort (n=12) in START: a completed, 24-month, open-label, single-arm, ascending-dose clinical trial of ZOLGENSMA.
bThis assessment is based on the Bayley Scales of Infant Development Version 3: Gross and Fine Motor Skills Subtexts, item 22, “Sits Without Support Series: 5 Seconds.”
cAlso includes patients who are observed sitting alone for ≥5, ≥10, or ≥30 seconds. Patients sitting ≥30 seconds are included in the total of ≥5 seconds (World Health Organization definition).
dThis assessment is based on the Bayley Scales: Gross and Fine Motor Skills Subtexts, item 26: “Sits Without Support Series: 30 seconds.”
e7/10 (70%) patients in the high-dose cohort are not currently receiving additional disease-modifying SMA treatment. 4 total patients in the high-dose cohort received nusinersen at any time in LTFU.