Key result
Onasemnogene abeparvovec systematic review evaluates efficacy and safety for spinal muscular atrophy type 1.
Supports onasemnogene abeparvovec in SMA1 care; extends gene therapy evidence in pediatric neuromuscular disease.
Our objective is to evaluate the efficacy and safety of onasemnogene abeparvovec in spinal muscular atrophy type 1 (SMA1) patients.
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Hanen et al. (2026) studied this question. Onasemnogene abeparvovec gene therapy was systematically reviewed to evaluate its therapeutic efficacy and safety in patients with spinal muscular atrophy type 1.
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