Your Voice is Needed Now!
Niemann-Pick UK is proud to stand with Genetic Alliance UK and the wider rare disease community in the Future for Rare campaign. This national initiative aims to ensure that the voices of patients, families, and carers directly shape the future of rare conditions policy in the UK.
The UK Rare Diseases Framework — which sets out priorities to improve diagnosis, care, and treatment for the millions of people living with rare conditions — has been extended to January 2027. This extension creates a vital opportunity to build on the existing framework with bolder ambitions, ring-fenced funding, stronger delivery teams, and genuine community involvement.
For families affected by Niemann-Pick diseases, the stakes are high. Many of us continue to face long diagnostic journeys, fragmented care, and barriers to accessing specialist treatments. The Future for Rare campaign is calling for real change in four key areas: • Faster, more accurate diagnosis • Greater awareness and training for healthcare professionals • Improved care coordination across services • Better access to specialised care and innovative treatments
How you can get involved right now:
- Complete the Future for Rare survey – open to anyone with lived experience of a rare condition, their families, carers, support organisations, healthcare professionals, and researchers. Survey link: https://www.smartsurvey.co.uk/s/future-for-rare/ Deadline: Friday 24 April 2026
- Submit evidence or policy recommendations via the campaign’s call for evidence.
- Sign up for campaign updates to hear about working groups, nationwide engagement events throughout 2026, and the national summit in September 2026.
Full campaign information, resources, and background documents (including the UK Rare Diseases Framework factsheet and “More than you can imagine” report) are available here: https://geneticalliance.org.uk/future-for-rare/
At Niemann-Pick UK we believe that when the rare disease community speaks with one powerful voice, real progress follows. Your experiences and insights are essential to making sure the next five years deliver meaningful improvements for every family living with Niemann-Pick diseases.
Take the survey today and help us build a brighter future for rare.
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