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Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial

Mohammad S, Bouchama A, Mohammad Alharbi B, Rashid M, Saleem Khatlani T, Gaber NS, et al

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Patients were, therefore, divided into two groups: positive fluid leakage (n = 10) and negative fluid leakage (n = 38)

This occurs in about 15% of children with NF1