Key result
Onasemnogene abeparvovec improved motor function, with a mean 11.0-point increase in CHOP-INTEND scores, and was well tolerated in children with spinal muscular atrophy type 1, including older and heavier patients.
Why the study?
Real-world data on the efficacy and safety of onasemnogene abeparvovec in SMA are needed, particularly to address uncertainties regarding its use in older and heavier children.
Does onasemnogene abeparvovec improve functional outcomes and is it safe in children with spinal muscular atrophy type 1?
Population
99 patients with genetically confirmed SMA type 1 treated with onasemnogene abeparvovec in the UK
Design
Multicentre observational cohort study
Follow-up
3–22 months
Authors
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Real-world OA data in older SMA type 1 patients support further study; leaves open need for randomized trials before practice change.
Cohort (n=99)
Yes
Does onasemnogene abeparvovec improve functional outcomes and is it safe in children with spinal muscular atrophy type 1?
Mean Difference: 11
Onasemnogene abeparvovec led to functional improvements and was well tolerated in children with SMA type 1, including older and heavier patients, though transaminitis and steroid requirements were higher in heavier patients.
Gowda et al. (2023) conducted a cohort in Spinal muscular atrophy type 1 (n=99). Onasemnogene abeparvovec was evaluated on Change in CHOP-INTEND score (MD 11.0). Onasemnogene abeparvovec improved motor function, with a mean 11.0-point increase in CHOP-INTEND scores, and was well tolerated in children with spinal muscular atrophy type 1, including older and heavier patients.
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