Overcoming the Next Barriers to Successful Therapy

Pediatr Endocrinol Rev. 2016 Jun:13 Suppl 1:629.
No abstract available

Publication types

  • Introductory Journal Article

MeSH terms

  • 1-Deoxynojirimycin / analogs & derivatives
  • 1-Deoxynojirimycin / therapeutic use
  • Administration, Oral
  • Cellulose
  • Daucus carota
  • Drug Delivery Systems
  • Drug Discovery
  • Enzyme Inhibitors / therapeutic use
  • Enzyme Replacement Therapy
  • Fabry Disease / drug therapy
  • Gaucher Disease / drug therapy
  • Humans
  • Lysosomal Storage Diseases / drug therapy*
  • Mucolipidoses / drug therapy
  • Pyrrolidines / therapeutic use
  • Rare Diseases / drug therapy*

Substances

  • Enzyme Inhibitors
  • Pyrrolidines
  • 1-Deoxynojirimycin
  • Cellulose
  • migalastat
  • eliglustat

Supplementary concepts

  • Neuraminidase deficiency with beta-galactosidase deficiency