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

Posts Tagged ‘Niemann-Pick’

NIH Clinical Trial of Cyclodextrin Update

NIH Clinical Trial of Cyclodextrin Update

TRND (Therapies for Rare and Neglected Diseases) and the NIH team met with FDA representatives on December 13th to discuss the progressive plans to bring Cyclodextrin to a clinical trial in 2012. For TRND, Niemann-Pick Type C is one of six pilot projects that were selected. The […]

Read more

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 […]

Read more

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.

Read more
Email Address First Name