SMA CLINICAL DATA, OUTCOME MEASURES AND REGISTRIES

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Economic considerations are increasingly important to help decision makers to efficiently allocate health care resources. To date, very little information is available on the cost-effectiveness of spinal muscular atrophy (SMA) treatment and newborn screening; yet more and more screening programs are being implemented in the USA and in Europe. The aim of this study is therefore to develop a decision-analytic model to assess the cost-effectiveness of newborn screening and pre-symptomatic treatment of SMA as compared to post-symptomatic treatment.

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