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A Rare Lysosomal Storage Disease
Contact: info@npcfund.org

Posts Tagged ‘NP-C’

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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Dementia in Our Kids – The Uninvited Thief

Dementia in Our Kids – The Uninvited Thief

Dementia, who was the thief that thought up this bright idea? Sorry honey, what did you say again? OK that might be a case of selective hearing. We’ve all have had family or known people who have suffered with this horrible symptom. DementiaWhen you mention dementia, Alzheimer’s comes to mind or we all tend to think of an elderly person with their hands on their head not able to remember out of frustration pounding the table with their fists. STOP!… WAIT!… Did you know our children can suffer from dementia too?

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Why Rare Diseases OUR Important

Why Rare Diseases OUR Important

How many times have you been looking for something at your house but you accidentally find something you previously were missing? Wouldn’t it be a shame if the prize you were seeking was within reach but you discounted that it could be that easy? What if understanding Niemann-Pick Type C disease opened up the door to help millions of Americans with other disorders involving cholesterol? Of course nothing in life is easy nor will it always be within reach. With being human comes the tendency to make oversights.

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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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New therapies – Niemann-Pick type C disease

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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