What the First Intrathecal Gene Therapy Means for SMA Clinicians and Researchers Marco Meglio

Published Date: 20 Dec 2025

Angela Lek, PhD, chief research officer of the Muscular Dystrophy Association, discusses the FDA approval of intrathecal onasemnogene abeparvovec (Itvisma; Novartis) and what it means for SMA treatment across older pediatric and adult populations.

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