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Treatment of infantile-onset spinal muscular atrophy with nusinersen: final report of a phase 2, open-label, multicentre, dose-escalation study

Dose-escalation StudySpinal Cord InjuryMedicineFinal ReportPediatric SpinePhase 2Neuromuscular PathologySpinal DisorderOrthopaedic SurgeryNeuromusculoskeletal Disorder