Efficacy and safety of onasemnogene abeparvovec for the treatment of patients with spinal muscular atrophy type 1: A systematic review with meta-analysis.
Brígida Dias FernandesBárbara Corrêa KrugFernanda D'Athayde RodriguesHérica Núbia Cardoso CiriloStéfani Sousa BorgesIda Vanessa D SchwartzLivia Fernandes ProbstIvan ZimmermannPublished in: PloS one (2024)
The results indicate a potential change in the natural history of type 1 SMA, but the methodological limitations of the studies make the real extent of the technology's long-term benefits uncertain.
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