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Description

This occurs in about 15% of children with NF1

What are the symptoms of JAS

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

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