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Newborn screening for spinal muscular atrophy with disease-modifying therapies: a cost-effectiveness analysis

28 Jul 2021

Abstract excerpt

OBJECTIVE: To assess cost-effectiveness of newborn screening (NBS) for spinal muscular atrophy (SMA) and early treatment with nusinersen or onasemnogene abeparvovec (gene therapy), compared with nusinersen without SMA screening. METHODS: Informed by an Australian state-wide SMA NBS programme, a decision analytical model nested with Markov models was constructed to evaluate costs and quality-adjusted life-years...

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PMID: 34321343
DOI: 10.1136/jnnp-2021-326344

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