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- W4380151480 abstract "Spinal muscular atrophy (SMA) is a rare neuromuscular disease caused by survival motor neuron (SMN) 1 gene mutation. Onasemnogene abeparvovec (ONA) is a single-administration gene therapy intended to be curative. We estimated the cost-effectiveness of ONA compared to best supportive care (BSC) in pre-symptomatic newborns diagnosed with SMA and two SMN2 copies—the most severe manifestation." @default.
- W4380151480 created "2023-06-11" @default.
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- W4380151480 date "2023-06-01" @default.
- W4380151480 modified "2023-09-26" @default.
- W4380151480 title "EE175 Cost-Effectiveness of Onasemnogene in Pre-Symptomatic Newborns with a Diagnosis of Spinal Muscular Atrophy and Two Copies of Survival Motor Neuron 2 Gene" @default.
- W4380151480 doi "https://doi.org/10.1016/j.jval.2023.03.476" @default.
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