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Preparing newborn screening for the future: a collaborative stakeholder engagement exploring challenges and opportunities to modernizing the newborn screening system

BACKGROUND AND OBJECTIVES: Projections that 60 transformative cell and gene therapies could be approved by the U.S. Food and Drug Administration (FDA) within 10 years underscore an urgent need to modernize the newborn screening (NBS) system. This study convened expert stakeholders to assess challeng...

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Detalles Bibliográficos
Autores principales: Andrews, Sara M., Porter, Katherine Ackerman, Bailey, Donald B., Peay, Holly L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8840788/
https://www.ncbi.nlm.nih.gov/pubmed/35151296
http://dx.doi.org/10.1186/s12887-021-03035-x