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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...

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Detalles Bibliográficos
Autores principales: Bailey, Donald B., Porter, Katherine Ackerman, Andrews, Sara M., Raspa, Melissa, Gwaltney, Angela Y., Peay, Holly L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Medical Association 2021
Materias:
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