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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...
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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Hematology
2022
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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 |
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