Cost-Effectiveness of Newborn Screening for Spinal Muscular Atrophy in The Netherlands

OBJECTIVESSpinal muscular atrophy (SMA) is a rare genetic disorder that causes progressive muscle weakness and paralysis. In its most common and severe form, the majority of untreated infants die before 2 years of age. Early detection and treatment, ideally before symptom onset, maximize survival an...

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Bibliographic Details

Published in:	Value in health Vol. 25; no. 10; pp. 1696 - 1704
Main Authors:	Velikanova, Rimma, van der Schans, Simon, Bischof, Matthias, van Olden, Rudolf Walther, Postma, Maarten, Boersma, Cornelis
Format:	Journal Article
Language:	English
Published:	01-10-2022
Online Access:	Get full text
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