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

Posts Tagged ‘NPC’

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16 Mar 2010

FDA says Hold Off on Zavesca (Miglustat) approval.

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Written by , Posted in Blog, News, Zavesca (Miglustat) | Leave a comment

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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13 Feb 2010

Social Security Adds 38 New Medical Conditions that Qualify for Disability

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Written by , Posted in Blog, News, Social Services | Leave a comment

Social Security adds 38 new medical conditions that qualify for disability help.  The new conditions range from early-onset Alzheimer’s disease to rare diseases that primarily affect children which includes Niemann-Pick Type C! Personally this seems like a relief for so many families. One of the goals of […]

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21 Nov 2009

New therapies – Niemann-Pick type C disease

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Written by , 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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