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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

01.11.21

Orphazyme provides regulatory update following Type A meeting with FDA on arimoclomol in Niemann-Pick disease type C

Progress made in understanding potential resolution of topics outlined in Complete Response Letter, including need for additional data to support NDA. FDA recommends the company provides supplemental information and analyses, and the FDA offers to have further interactions to identify a path to resubmission for arimoclomol in NPC...

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