New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat

@inproceedings{Wraith2009NewTI,
  title={New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat},
  author={James Ed Wraith and Jackie Imrie},
  booktitle={Therapeutics and clinical risk management},
  year={2009}
}
Niemann-Pick disease type C (NP-C) is an autosomal recessive disorder characterized by progressive neurological deterioration leading to premature death. The disease is caused by mutations in one of two genes, NPC1 or NPC2, leading to impaired intracellular lipid transport and build-up of lipids in various tissues, particularly the brain. Miglustat (Zavesca(R)), a reversible inhibitor of glycosphingolipid synthesis, has recently been authorized in the European Union, Brazil and South Korea for… CONTINUE READING

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