Key result
Onasemnogene abeparvovec improved CHOP-INTEND scores significantly at 3 months for patients <24 months old and at 12 months for those >24 months, with adverse events occurring in 51 of 67 patients.
Why the study?
Clinical trials of onasemnogene abeparvovec were limited to spinal muscular atrophy infants under 7 months and <8.5 kg, leaving efficacy and safety predictors unexamined across wider age and weight ranges and after switching from other therapies.
Does onasemnogene abeparvovec improve motor function and what is its safety profile in a broad age and weight range of patients with spinal muscular atrophy?
Population
67 spinal muscular atrophy patients aged 22 days-72 months and 3.2-17 kg
Comparison
Onasemnogene abeparvovec in treatment-naïve patients vs those previously treated with other drugs
Follow-up
12 months
Authors
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Extends OA data to wider SMA age/weight and pretreated patients; leaves open need for controlled trials before practice change.
Cohort (n=67)
Does onasemnogene abeparvovec improve motor function and what is its safety profile in a broad age and weight range of patients with spinal muscular atrophy?
Onasemnogene abeparvovec demonstrates efficacy across broader age and weight groups than those in initial clinical trials, with age at treatment serving as a key predictor for both motor function improvement and risk of transaminase elevation.
Pane et al. (2023) conducted a cohort in Spinal muscular atrophy (n=67). Onasemnogene abeparvovec was evaluated on CHOP-INTEND changes. Onasemnogene abeparvovec improved CHOP-INTEND scores significantly at 3 months for patients <24 months old and at 12 months for those >24 months, with adverse events occurring in 51 of 67 patients.
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