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Research

Alongside providing support and care for those living with or affected by Niemann-Pick disease, one of our other main areas of focus is providing information relating to research. NPUK shares knowledge, expertise and works in partnership and collaboration with other not-for-profit organisations, medical authorities and scientific institutions across the world, in order to facilitate progress towards therapeutic interventions for the Niemann-Pick disease.

Where possible, NPUK also funds appropriate research in order to improve medical and scientific knowledge and promote understanding of the disease. The results are often shared amongst our community of families and health professionals at the Annual Family Conference & Interactive Workshop we hold every September. It is through this medical research and clinical trials that we will find innovative new ways in which to better manage Niemann-Pick disease.

Research

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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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