Novartis's Gene Therapy Zolgensma Has Durable Effects At Five Years
Intrathecal Therapy's 'Remarkable' Disease Scores
Executive Summary
AveXis/Novartis’s one-time AAV9 vector-based intravenous gene therapy for spinal muscular atrophy, Zolgensma, continues to have beneficial effects up to five years after its administration to babies, and an intrathecal formulation has significant promise in older infants.
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