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

Posts Tagged ‘Cholesterol’

Orphan Drug Designation for Cyclodextrin | NPC

This week the FDA has stated formally that they see promise in the use of Cyclodextrin in treating Niemann-Pick Type C (NPC) by granting its “Orphan Drug Designation”. In January of 1983, the Orphan Drug Act (ODA) was passed into law in the United States with consistent […]

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Why refer to it as the “Childhood Alzheimer’s”?

Why refer to it as the “Childhood Alzheimer’s”?

You possibly can recall someone you knew that suffered from Alzheimer’s disease. Currently 5.3 million Americans are living with this progressive and fatal brain disease with no cure (www.Alz.org). Alzheimer’s is progressively devastating in how it destroys brain cells, causes loss of memory (progressive dementia), and problems […]

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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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