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Benefits of newborn screening and hematopoietic cell transplant in infantile Krabbe disease

Infantile Krabbe disease (IKD) can be treated with hematopoietic cell transplantation (HCT) if done during the first weeks of life before symptoms develop. To facilitate this, newborn screening (NBS) has been instituted in 8 US states. An application to add IKD to the recommended NBS panel is curren...

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Detalles Bibliográficos
Autores principales: Page, Kristin M., Ream, Margie A., Rangarajan, Hemalatha G., Galindo, Rafael, Mian, Ali Y., Ho, Mai-Lan, Provenzale, James, Gustafson, Kathryn E., Rubin, Jennifer, Shenoy, Shalini, Kurtzberg, Joanne
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Hematology 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9092415/
https://www.ncbi.nlm.nih.gov/pubmed/35042231
http://dx.doi.org/10.1182/bloodadvances.2021006094