Newborn Screening for Spinal Muscular Atrophy and Lysosomal Storage Disorders Takes Advantage of Novel Therapies

J Pediatr. 2017 Nov:190:9-10. doi: 10.1016/j.jpeds.2017.08.016.
No abstract available

Keywords: Fabry; Gaucher; MPS-I; Niemann-Pick; Pompe; lysosomal; newborn screening; spinal muscular atrophy; storage disorder.

Publication types

  • Editorial
  • Comment

MeSH terms

  • Dried Blood Spot Testing
  • Humans
  • Infant, Newborn
  • Lysosomal Storage Diseases*
  • Muscular Atrophy, Spinal
  • Neonatal Screening*