START: An open-label, single-arm, ascending-dose clinical trial demonstrated the efficacy of ZOLGENSMA1,2

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.1
bNatural history: The Pediatric Neuromuscular Clinical Research (PNCR) Network study population, 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.3
cHigh-dose cohort (n=12) in a 24-month clinical trial of ZOLGENSMA.

All 12 patients in the high-dose cohort were alive and free of permanent ventilation at 24 months post infusion1,2,4

START: Patients from the high-dose ZOLGENSMA cohort achieved key motor milestones

Milestones were achieved and maintained with ZOLGENSMA in patients from the high-dose cohort2,4

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.”

START: ZOLGENSMA resulted in rapid and sustained motor function improvements from baseline2,5

In the high-dose cohort, ZOLGENSMA significantly improved motor function as early as 1 month post infusion2,5

CHOP INTEND=Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders
aHigh-dose cohort (n=12) in START: a completed, 24-month, open-label, single-arm, ascending-dose clinical trial of ZOLGENSMA.
bScores on the CHOP INTEND scale of motor function range from 0 to 64, with higher scores indicating better function.7

Early intervention was correlated with improved outcomes5

Understand the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND)

CHOP INTEND was specifically developed to assess motor function in patients with SMA. The CHOP INTEND score is measured in points, which are assigned based on a patient’s ability to perform specific motor skills. The test consists of 16 measures, each scored on a scale 0-4, with the highest possible score being 64 points.7

START: ZOLGENSMA helped maintain respiratory and nutritional status in patients from the high-dose cohort who were free of support prior to infusion2

BiPAP=Bi-level positive airway pressure.
aHigh-dose cohort (n=12) in START: a completed, 24-month, open-label, single-arm, ascending-dose clinical trial of ZOLGENSMA.
bOne additional patient with no BiPAP prior to gene therapy continues without regular BiPAP, but uses when ill.2

START: ZOLGENSMA helped sustain bulbar function in patients from the high-dose cohort2

aHigh-dose cohort (n=12) in START: a completed, 24-month, open-label, single-arm, ascending-dose clinical trial of ZOLGENSMA.
bSwallow function in patients were last assessed at 24 months, however, one patient had the last assessment at 12 months.2