A Rare Lysosomal Storage Disease

Posts Tagged ‘Zavesca’

Canada Receives First Approved Treatment For Niemann-Pick Type C

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 […]

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FDA says Hold Off on Zavesca (Miglustat) approval.

On March 9, 2010 the FDA has requested additional preclinical and clinical information regarding Zavesca (Miglustat) before giving the ultimate approval that the Niemann-Pick Type C community was hoping for.

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UPDATE 2-US FDA panel backs new use for Actelion drug [Zavesca]

We’re a few weeks behind in updating this information but some big steps have happened recently regarding the potential FDA approval for Zavesca. The US Advisory panel on Jan. 12th recommended approval! This snippet is from the article on Reuters regarding the breaking news: Doctors can prescribe […]

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Historic FDA related drug advisory committee will meet | Zavesca

This is an email from Nadine Hill of the NNPDF. This is good news. If the FDA does say lets move forward that means more families might be able to participate because the drug would be FDA approved. All though this isn’t a cure it is one […]

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