Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial - Nature Medicine

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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