Article
Acceptability of newborn screening for Spinal Muscular Atrophy: views of the UK public, screened families, health professionals and SMA community
2026-02-23
Abstract excerpt
<title>Abstract</title> <p>With the advent of novel gene therapies, rare genetic diseases once lacking treatments are now being considered for newborn screening programmes (NBS). Indeed, Wilson and Jungner criteria (which are drawn on worldwide to guide screening programme evaluation) necessitate treatment availability for a recommendation of screening. Spinal muscular atrophy (SMA) is an example of a condition f...
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Identifiers and source
- Literature Corpus work
- ea1b3665-37d2-5dc2-b0bf-051b1257f278
- DOI
- 10.21203/rs.3.rs-8881933/v1
