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Go Make Memories

NPUK Short Film: Go Make Memories

We are proud of our collaborative short film Go Make Memories (2019), which has helped push for further awareness of Niemann-Pick disease and gained many awards in the process. Please take a moment to watch and share on our YouTube channel @NPUK

NPUK Annual Family Conference & Interactive Workshop

Digital Annual Family Conference & Interactive Workshop

Our Digital Annual Family Conference & Interactive Workshop can now be accessed "on-demand" on the WorkCast platform for those wishing to watch and/or re-watch our milestone event online! If you have any issues with accessing the site please do get in touch with us by email at: info@npuk.org or phone on: 0191 415 06 93

NPUK

NPUK Support Fund

The NPUK Support Fund is still in place to help individuals and families within the NPUK Community. The aim of this limited fund is to assist those experiencing hardship or unexpected expense as a result of the Coronavirus pandemic, which threatens to impact their physical or mental well-being. Find out more information at: npuk.org/npuk-2020-support-fund

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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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