Filmmaker Paul Stainthorpe has spent a decade telling emotionally challenging stories, but after serving as Assistant Director on two Niemann-Pick awareness films: Hadid and I (2025) and Never Heard of It (TBC), he is set to do something new – lead his very own awareness project on behalf of Niemann-Pick UK… Paul Stainthorpe is a […]
We’re celebrating ten years of John Lee Taggart at Niemann-Pick UK. John joined the team in a more junior communications role and has grown into the Head of Communications we know today. Along the way he’s led films, campaigns, animations, publications and international projects — always with the same quiet focus on helping families feel […]
This week, Niemann-Pick UK (NPUK) and The International Niemann-Pick Disease Alliance (INPDA) met with Zevra following the disappointing CHMP opinion on the EU marketing authorisation for arimoclomol. Zevra reaffirmed its commitment to the NPC community and its intention to request a re-examination. Further updates will be shared when available. Zevra’s press release: https://investors.zevra.com/news-releases/news-release-details/zevra-therapeutics-provides-update-regulatory-submission EMA letter (attached) […]
On 9 July 2026 a group of scientists from the University of Oxford and University College London set off from the Department of Pharmacology in Oxford for what would become their toughest challenge yet: cycling approximately 130 miles to Stonehenge in Wiltshire and back over two days. Riders: Fran Platt, Ahad Rahim, Danielle Taylor-te Vruchte, […]
The 20th Annual NPUK Golf Day, July 2026 Our 20th Annual Niemann-Pick UK Golf Day at Ramside Hall Golf Club was a powerful reminder of what community, compassion and shared purpose can achieve. The venue itself felt renewed – fresher, brighter and more welcoming than in previous years, adding an extra layer of joy to […]
19th – 21st June 2026 Wearside Golf Club hosted its annual Captain’s Weekend in June 2026, delivering three days of golf, laughter, friendly competition and community spirit. The weekend featured three different competitions each day, with a wide range of fun prizes on offer. Members and guests enjoyed the course in good weather, shared plenty […]
Helping to improve understanding and earlier diagnosis through lived experience Families affected by Niemann-Pick often experience a long and complex journey before receiving a diagnosis. Understanding these rea-life experiences is key to improving awareness and supporting earlier diagnosis in the future. My Rare Journey is a platform created by OpalMedica to collect diagnostic stories from […]
We want to say a heartfelt thank you to Dave Smith for all of his support for Niemann-Pick UK, enjoy your well-deserved retirement. The entire Staff and Board of Niemann-Pick UK (NPUK) want to express our sincere gratitude to the wonderful Dave Smith, former Research Assistant at the Department of Pharmacology (University of Oxford), for […]
A Legacy of Love and Fundraising Every year, a group of golfers gather for a day filled with swings, smiles, and a shared purpose that goes beyond the fairways. The Annual Niemann-Pick UK (NPUK) Golf Day, organised by Craig Mathieson, is more than just a sporting event—it’s a heartfelt tribute to his niece, Lucy Mathieson, […]
Without Xenpozyme, Caitlin Monachan’s rare genetic disorder may kill her. But she must wait for the medication to be approved in the UK Early last year, 26-year-old Caitlin Monachan received a devastating diagnosis. She has Niemann-Pick disease, an ultra-rare genetic disorder caused by a missing enzyme, where toxic material builds up in the body, fatally damaging the lungs, liver […]
