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

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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Bibliographic Details
Main Authors: Sophy T. F. Shih, Elena Keller, Veronica Wiley, Michelle A. Farrar, Melanie Wong, Georgina M. Chambers
Format: Article
Language:English
Published: MDPI AG 2022-07-01
Series:International Journal of Neonatal Screening
Subjects:
SMA
SCID
newborn screening
cost-effectiveness
Markov model
budget impact analysis
Online Access:https://www.mdpi.com/2409-515X/8/3/45

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https://www.mdpi.com/2409-515X/8/3/45

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