Major Developmental Milestones Met in Infants, Zolgensma Data Show

Major Developmental Milestones Met in Infants, Zolgensma Data Show

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Major Developmental Milestones Met in Infants, Zolgensma Data Show

New data about Zolgensma, the one-time gene therapy for children with spinal muscular atrophy (SMA), demonstrate age-appropriate development when used pre-symptomatically, and rapid, meaningful efficacy in symptomatic children, even those with severe disease before treatment, according to two Phase 3 clinical trials. The therapy, developed by Novartis, uses a harmless virus to deliver a functional copy of SMN1, the gene mutated gene in SMA. A lack of this gene results in the rapid loss of motor…

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