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  • Shih-hsin Kan, Mika Aoyagi-Scharber, +27 authors Elizabeth F. Neufeld
  • Medicine, Biology
  • Proceedings of the National Academy of Sciences
  • 2014 (First Publication: 29 September 2014)
  • Significance Mucopolysaccharidosis type IIIB (MPS IIIB) is a devastating and currently untreatable disease affecting mainly the brain. The cause is lack of the lysosomal enzyme,Expand
  • Priya S. Kishnani, Patricia I. Dickson, +18 authors Anne R. Pariser
  • Medicine
  • Molecular genetics and metabolism
  • 2016 (First Publication: 1 February 2016)
  • The US Food and Drug Administration (FDA) and National Organization for Rare Disease (NORD) convened a public workshop titled "Immune Responses to Enzyme Replacement Therapies: Role of ImmuneExpand