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Modelling the Cost-Effectiveness and Budget Impact of a Newborn Screening Program for Spinal Muscular Atrophy and Severe Combined Immunodeficiency

Spinal muscular atrophy (SMA) and severe combined immunodeficiency (SCID) are rare, inherited genetic disorders with severe mortality and morbidity. The benefits of early diagnosis and initiation of treatment are now increasingly recognized, with the most benefits in patients treated prior to sympto...

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Detalles Bibliográficos
Autores principales: Shih, Sophy T. F., Keller, Elena, Wiley, Veronica, Farrar, Michelle A., Wong, Melanie, Chambers, Georgina M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9326684/
https://www.ncbi.nlm.nih.gov/pubmed/35892475
http://dx.doi.org/10.3390/ijns8030045