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Research

Alongside providing support and care for those living with or affected by Niemann-Pick disease(s), 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 Niemann-Pick diseases.

Where possible, NPUK also funds appropriate research in order to improve medical and scientific knowledge and promote understanding of the diseases. The results are often shared amongst our community of families, friends and health professionals at the Annual Family Conference & Interactive Workshop we hold every September (2020 dates TBC due to COVID-19 pandemic). It is through this medical research and clinical trials that we will find innovative new ways in which to better manage Niemann-Pick diseases.

Research

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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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