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PM Society Digital Awards 2020

We are pleased to announce earlier in the year that we were nominated in two categories for this year's prestigious PM Society Digital Awards 2020 for our work on our latest short film, Go Make Memories (Carl Mason, 2019). We have since scooped awards in both categories, a Silver in the Effectiveness: Healthcare Charities & Patient Associations group, and Gold in Craft: Film. Thank you PM Society, we are truly honoured!

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: or phone on: 0191 415 06 93

Invisible Illnesses (Upcoming Campaign)

We are currently developing an awareness campaign entitled "Invisible Illnesses", which will focus primarily on ASMD Niemann-Pick disease type B, but will be open to all members of the NPUK Community with scope for collaboration with the wider rare disease population too! If you feel you could contribute from this campaign please get in touch with us at:

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Latest news


MNK Announcement Jan 2021

We have received the following statement from Mallinckrodt Pharmaceuticals, stating their intention to discontinue clinical development of adrabetadex (VTS-270) for Niemann-Pick Type C1 disease (NPC), effective immediately. We understand that this will be very difficult news for many within our community; the NPUK team will be available if you would like to contact us: email or telephone 0191 415 06 93.

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UK Disability Survey

The government has just announced a new UK Disability Survey and we want to hear from as many people as possible. They are particularly keen to hear from disabled people, their carers, friends and family but views from the wider public are also very welcome. The survey will remain open until 23rd April, and all views will be used to shape the delivery of the plans we set out in the Strategy with those that we receive by 13th February informing its development.

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