Newborn Screening for Krabbe Disease and Other Lysosomal Storage Disorders: Broad Lessons Learned

Newborn screening (NBS) for Krabbe disease (KD) began in New York (NY) in August 2006. In summary, after eight years of screening there were five infants identified with early-onset Krabbe disease. Four underwent transplant, two are surviving with moderate to severe handicaps, and two died from tran...

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Bibliographic Details
Main Authors: Joseph J. Orsini, Michele Caggana
Format: Article
Language:English
Published: MDPI AG 2017-03-01
Series:International Journal of Neonatal Screening
Subjects:
Online Access:http://www.mdpi.com/2409-515X/3/1/3