Zolgensma® (onasemnogene abeparvovec-xioi) – New orphan drug approval
May 24, 2019, the FDA announced the approval of Novartis/AveXis’ Zolgensma (onasemnogene abeparvovec-xioi), for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
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