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Without people like you getting involved as a volunteer, fundraiser, or anything in between - we wouldn't be able to provide the care and support that we provide to families affected by Niemann-Pick disease! There are numerous opportunities available when it comes to helping us - the only limit is your imagination; there's everything from taking part in our fun days as a member of our passionate volunteer team, setting up fundraisers, or drumming up interest and donations through creative sponsored events. By working together we can make a real difference to the lives of those people affected by Niemann-Pick disease. Please contact us for more information on how you can join us, by email at: info@npuk.org or by phone on: 0191 415 06 93.

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Latest news

29.11.19

#ChildrenAtTheHeart

We are joining 140+ organisations in a call to all political parties to put children and young people at the heart of this General Election...

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19.11.19

Orphazyme receives Breakthrough Therapy Designation for arimoclomol in Niemann-Pick Disease Type C (NPC)

Kim Stratton, Chief Executive Officer at Orphazyme, commented, “We are delighted with FDA’s decision to grant Breakthrough Therapy Designation to arimoclomol for NPC. Arimoclomol has been shown to have a clinically meaningful effect on disease progression in NPC that is further supported by a biomarker effect indicating an effect on the biological underpinnings of the disease and a favorable safety and tolerability profile. We are committed to bringing this product to patients as soon as possible. Breakthrough Therapy Designation is designed to expedite the development and review of products for serious diseases with the direct involvement of senior staff and we look forward to working closely with the FDA to further advance arimoclomol. Our preparations for filing in the US are underway and we are on track to submit a New Drug Application in H1 2020.”

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