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Novartis’ $2 Million Gene Therapy Zolgensma Shows ‘Remarkable’ Results and Offers Hope for Children with SMA

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With a price tag of over $2.5 The prevalence of SMA is about one in 10,000 children, and one in 54 people carry the SM1 genetic mutation; two carriers have a 25 percent chance of having a child with SMA. million, Zolgensma is currently the world’s most expensive drug. Related: Is $2 Million Too Much For FDA-Approved SMA Gene Therapy?