Fig 1.
Flowchart of studies’ selection.
Adapted from: Page et al., 2021 [19]. Available at: http://www.prisma-statement.org/.
Table 1.
Characterization of studies included in the systematic review.
Fig 2.
Risk of bias assessment, outcomes of global survival and mechanical-ventilation-free survival (A), improvement in motor function (B) and treatment-related adverse events (C).
Fig 3.
Meta-analysis of a) overall survival, b) event-free survival and c) CHOP-INTEND ≥ 40 following administration of onasemnogene abeparvovec in SMA type 1 patients.
Fig 4.
Meta-analysis of a) serious adverse events and b) drug-related adverse events following administration of onasemnogene abeparvovec in SMA type 1 patients.
Table 2.
Profile of certainty of evidence in the treatment with onasemnogene abeparvovec in patients of up to 2 years of age diagnosed with SMA type 1.