Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapies.

EClinicalMedicine · 2023 · PMC10184045 · PMID 37197706

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did not reach statistical significanceno p-value reported

Thrombocytopenia was more frequent in older and heavier patients and in those previously treated but this did not reach statistical significance.

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