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About Us

Niemann-Pick UK (NPUK) is a charitable organisation dedicated to making a positive difference to the lives of those affected by Niemann-Pick diseases. We raise much needed awareness, provide practical and emotional support, advice and information and facilitate research into potential therapies.

We are a small charity with less than five hundred members, the work we do is funded entirely through fundraising activities and successful grant applications. Our strength, therefore, comes from the dedication of our much valued families, members, benefactors and volunteers who work with tremendous effort and loyalty in support of the charity.

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Latest news

11.01.19

CTD Announces Presentation at Upcoming Conference on Lysosomal Storage Diseases

CTD Holdings: Annual conference offers opportunities for academic researchers and industry representatives to exchange information on novel therapeutic interventions...

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08.01.19

Sanofi Genzyme: ASCEND and ASCEND-Peds Studies Complete Enrollment

Sanofi Genzyme’s Global Project Team would like to announce that our pivotal registration study (ASCEND Study) for adult patients with chronic, systemic acid sphingomyelinase deficiency (ASMD) Types B and A/B (or intermediate type) and our pediatric study (ASCEND-Peds) also treating Type B and A/B ASMD pediatric patients have successfully completed enrollment for the targeted number of patients for each of these trials...

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