Latest News from NPUK

NPUK has been informed by Sanofi of supply interruptions affecting Xenpozyme (olipudase alfa), a treatment for Acid Sphingomyelinase Deficiency (ASMD). As of November 2026, Sanofi anticipates a shortage of olipudase alfa in some countries. Sanofi has also advised that, based on current supply forecasts and anticipated consumption, global supply of olipudase alfa is expected to […]

October 29, 2026 Dear NPC Organizational Leaders, As we approach the conclusion of Niemann-Pick Disease Awareness Month, we are overjoyed to announce the submission of our New Drug Application (NDA) to the FDA for Trappsol® Cyclo™ (remibetadex, previously identified as intravenous hydroxypropyl-β-cyclodextrin). This NDA seeks approval for the treatment of the neurovisceral manifestations of NPC […]

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 […]

On 5 October 2026, IntraBio announced that it has entered into an agreement to be acquired by Shionogi, a global pharmaceutical company with a growing commitment to rare disease. We recognise that news like this may naturally raise questions for people living with Niemann-Pick disease type C (NPC), their families and the wider community. We […]

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 […]

Longitudinal study includes first published real-world data on arimoclomol in adults with NPC and provides insights across a broad patient population BOSTON, Aug. 03, 2026 (GLOBE NEWSWIRE) — Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on bringing life-changing therapeutics to people living with rare diseases, today announced the publication […]

BOSTON, June 23, 2026 (GLOBE NEWSWIRE) — Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on bringing life-changing therapeutics to people living with rare diseases, announced the publication of “Arimoclomol in infants with Niemann-Pick disease type C: Results from the phase 2/3 open-label pediatric substudy” in Molecular Genetics and Metabolism […]

A Significant Step Forward in NPC Research We’re pleased to share an important update from Cyclo Therapeutics, marking a significant moment in ongoing research into Niemann-Pick disease type C (NPC) and offering a real sense of progress for the global community affected by this rare condition. Cyclo Therapeutics has announced that the final patient has […]

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: […]