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Expert Evaluation of Strategies to Modernize Newborn Screening in the United States
IMPORTANCE: Novel therapies, including cell and gene therapies, can radically improve outcomes among patients with rare disorders, especially if provided early. Newborn screening (NBS) could support early access to novel therapies, but the speed of new therapy development is a disruptive event for w...
Autores principales: | , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Medical Association
2021
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8717100/ https://www.ncbi.nlm.nih.gov/pubmed/34964853 http://dx.doi.org/10.1001/jamanetworkopen.2021.40998 |