A Rare Lysosomal Storage Disease

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Posts Tagged ‘Miglustat’

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10 Apr 2010

Canada Receives First Approved Treatment For Niemann-Pick Type C

Tags: Actelion, Canada, Canadian Chapter of the National Niemann Pick Disease Foundation, CCNNPDF, Gaucher's Disease, Health Canada, Jean-Paul Clozel M.D. CEO, Michael G. Stults, Miglustat, Montreal Children Hospital, Niemann-Pick Type C Disease, NP-C, NPC, Serge Melancon M.D., Zavesca
Written by Michael G Stults, Posted in Blog, News, Zavesca (Miglustat) | 1 Comment

Canada has a lot to celebrate for their current and future patients living with the “Childhood Alzheimer’s” or Niemann-Pick Type C (NPC). During the last full week of March 2010, the announcement came down that Health Canada [The Food Directorate, the Therapeutic Products Directorate and the Natural […]

21 Nov 2009

New therapies – Niemann-Pick type C disease

Tags: Jackie Imrie, James E Wraith, Manchester, Miglustat, Niemann-Pick Type C Disease, NP-C, NPC, Research, Royal Manchester Children’s Hospital, Therapies, UK, Willink Biochemical Genetics Unit, Zevesca
Written by Michael G Stults, Posted in Blog, News, Therapies, What is NPC | Leave a comment

Abstract: Niemann-Pick disease type C (NP-C) is an autosomal recessive disorder characterized by progressive neurological deterioration leading to premature death.

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