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

• Prif Awduron: Joseph J. Orsini (Awdur), Michele Caggana (Awdur)
• Fformat: Llyfr
• Cyhoeddwyd: MDPI AG, 2017-03-01T00:00:00Z.
• Pynciau: article
• Mynediad Ar-lein: Connect to this object online.

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...