Information for families and the NPC community We know that people living with Niemann-Pick disease type C (NPC), and their families, want to be kept informed about research and potential new treatment opportunities. We would therefore like to share information about NAVIGATE, a clinical trial that is currently recruiting children and adults with NPC. Please […]
As we begin Niemann-Pick Disease Awareness Month, we are shining a spotlight on the incredible efforts of families, friends and supporters who are helping to raise awareness of this rare disease and make a difference to those affected by it. To start the month, we are delighted to share the story of Alfie Lewis, who […]
At Niemann-Pick UK, we know that the road to an ASMD diagnosis is often not straightforward. Many individuals and families experience years of uncertainty, unanswered questions, and repeated visits to healthcare professionals before finally receiving a diagnosis. By sharing your story, you can help us better understand these experiences and identify what might help others […]
National Lottery Community Fund Supports Innovative Projects to Amplify Patient Voices Across Wales and Northern Ireland We are delighted to share some really positive news…we’ve actually been awarded TWO grants by the National Lottery Community Fund! The first is a 12-month Wales-based communications programme focused on co-producing accessible video resources. The second is the Niemann-Pick […]
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 […]
What is Childhood Dementia? Childhood dementia is an area that is under recognised and often misunderstood, despite there being over 140 neurodegenerative conditions – including Niemann-Pick disease type C – that can cause it. As these conditions progress, families face daily challenges in providing complex, round-the-clock care. The impact on family life is significant: […]
Help Shape the Future: Survey on Living with Childhood Dementia Niemann-Pick UK (NPUK) is dedicated to supporting individuals and families affected by Niemann-Pick diseases, a group of rare lysosomal storage disorders that can lead to childhood dementia, among other symptoms. We are proud to promote the International Niemann-Pick Disease Registry (INPDR) survey on the “Lived […]
Rebecca French’s Research on Niemann-Pick Community Perspectives on Cure vs. Prevention in Rare Diseases Rebecca French, a fourth-year student at the University of Bath, is conducting research titled “What are the Niemann-Pick Community in England’s perspectives and policy implications of investing in gene therapy cures versus pre-emptive genetic testing for rare diseases?” This ethics-approved study […]
Urgent Appeal for Support After Malicious Attack on September 11th On the night of September 11th, a malicious fire completely destroyed Vermont House, the long-standing base for Niemann-Pick UK (NPUK)’s Central Office. This catastrophic attack has left our charity facing immense challenges, with the entire building reduced to ruins. Caught in the crossfire of ongoing […]