For infants with three copies of SMN1 at risk for SMA type 1, onasemnogene abeparvovec improves ventilator-free survival and nutritional/respiratory independence and allows motor development similar to healthy children without SMA. 2022SMAConference
gene modifier variant would not be included in the ITT population. Efficacy and safety findings for children with threeSMN2
who met the ITT criteria and were ≤6 weeks of age at the time of gene replacement therapy . Full eligibility criteria are described in the Supplementary Material. The Coronavirus Disease 2019 pandemic did not affect retention. No participant withdrew from SPR1NT or was lost to follow-up because of the COVID-19 pandemic. However, some scheduled study visits and assessments were delayed or cancelled because of restrictions caused by the COVID-19 pandemic.All children were admitted into the hospital for pretreatment baseline procedures 1 day before infusion. For all assessments, baseline was defined as the last assessment conducted before dosing.
Outpatient follow-up assessments were conducted on Days 7, 14, 21, 30, 44, 51 , 60, and 72 post-dose, and then at 3 months of age and every 3 months thereafter through 24 months of age . All eligible children were invited to enroll in an ongoing long-term follow-up study .The primary efficacy endpoint was the ability to stand independently for ≥3 seconds at any visit up to 24 months of age, as stipulated by item #40 from the gross motor subtest of the BSID.
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