‘Best Possible Outcome’ for SMA? Newborn Screening, Then Zolgensma

‘Best Possible Outcome’ for SMA? Newborn Screening, Then Zolgensma

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‘Best Possible Outcome’ for SMA? Newborn Screening, Then Zolgensma

Despite its high cost, giving the one-time gene therapy Zolgensma to an infant identified through newborn screening with spinal muscular atrophy (SMA) is cheaper in the long run than starting treatment with Spinraza (nusinersen) after symptom onset, according to a cost-effectiveness study in Australia. Combining newborn screening (NBS) and early use of Zolgensma was not only associated with lower overall costs, but also with longer and better lives for these children. “Pairing newborn screening with early gene therapy…

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