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

Archive for the ‘What is NPC’ Category

Impact of Niemann-Pick Type C

Actelion Pharmaceuticals LTD produced a short video on two families and how Niemann-Pick Type C Disease has impacted their lives. Hollie Carter and Annie Pyne are featured in this video. They both are youngsters living with this disease. At first sight we all instantly start to evaluate […]

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What is Niemann-Pick Type C?

What is Niemann-Pick Type C Disease? View more presentations from Michael G. Stults.

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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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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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