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For the first time in the UK, in collaboration with the NIHR, GOSH and the BRC we brought together families affected by BPAN to hear the latest updates on all things research and charity. Around 140 people, mainly from across the UK, but also with families joining us from Greece, Spain, Italy, USA and Latvia, others dialling in from Australia, Ukraine and Mexico. The feedback from families has been overwhelmingly positive. Feeling supported, connected and understood, and for the first time, truly part of something that is making a difference. Many are already asking how they can get involved and that says everything! One of the most important parts of the day was bringing the research to families. Hearing directly from the teams at Great Ormond Street Hospital and University College London, learning about the latest progress in gene therapy and cardiac glycosides, and having the chance to ask the questions that really matter. The researchers themselves left feeling more motivated than ever. Meeting so many incredible children and families had a real impact, reminding them exactly why this work matters and strengthening their drive to push the science forward even harder. When we met these incredible researchers, for the first time we felt real hope. That’s what we wanted to bring to as many families as we could, the knowledge that this is a very real opportunity to get behind and support. The commitment, the expertise, the belief… it’s all there. The reality is, this comes down to funding that can unlock life-changing treatments and real improvements in quality of life for people living with BPAN. This day was nearly a year in the making, and it simply wouldn’t have happened without the incredible support behind the scenes, from Great Ormond Street Hospital and Charity, UCL, NIHR Biomedical Research Centre and The National Lottery Community Fund, to the researchers, volunteers, our BPAN Unites charities, BPAN Heroes and BPAN Matters and every single family who joined us, in person or online. An extra special thank you to our family presenters Lindsey Knight & Laura for being so brave and vulnerable in sharing their lived experiences with their children Thank you for showing what’s possible when we come together like this. Because alongside the science, something else has now been built. A community that supports each other. A community that understands. A community that knows we are not alone. Stronger together, always. 🩷💙

We thrilled to announce first ever UK BPAN Family Day is open for registration! We invite you to join us in London for a dedicated in-person event bringing families and researchers together to exchange knowledge, build connections, and help shape the future of BPAN research. Organised in partnership with Great Ormond Street Hospital, the NIHR Biomedical Research Centre (BRC), and UCL. This day is about listening to families, strengthening understanding, and ensuring patient voices help inform the direction of research now and in the future! See full details & itinerary for the day here: https://www.gosh.nhs.uk/.../support.../brc-events/bpan-day/ We’re incredibly proud to bring this long overdue day to you alongside the World-class and super committed clinical & research teams Please DM or Email us if you have any questions engage@actionforbpan.co.uk

Today, 28th January 2026 we were invited the BRC/NIHR GOSH Gene Therapy in Paediatrics event to represent our small but determined community. The day focused on bringing together GPs, paediatricians, clinicians and patient advocacy groups to provide an understanding of gene therapy technologies, long term effects, latest advancements & the future of gene therapy.  For us, the most impactful part of the day was hearing from the patients and their families who’ve received gene therapy and the incredible benefits that have been truly life-changing 🩷💙 We’re grateful to be part of these conversations and to ensure BPAN families are seen, heard and represented. We even bumped into one of our own BPAN Gene Therapy Professors Simon Waddington!

Action For BPAN Weekly Lottery Your Chance to WIN £25,000 Each £1 entry helps Action for BPAN fund vital research and provide essential support, bringing us all one step closer to a cure. Where else could £1 do so much? Play Now As well as the chance to win big, 50p of every £1 played in the lottery supports Action for BPAN to fund critical research into BPAN and support families living with this rare and life-limiting condition. Your entry helps bring us closer to better understanding BPAN, developing treatments, and ultimately a cure. It also ensures families have access to the support, information, and community they need, so no one faces BPAN alone. Its Easy to Play It’s really simple to get started, all you need to do is enter your details, choose the way you want to pay and then select your number of entries. It costs just £1 per entry. Submit your details and we will send you your lucky numbers. For every £1 you play each week, you’ll receive a unique 6-digit lottery number. Winning numbers are revealed each week, match your numbers to win up to £25,000. Every Friday a winning number is drawn at random. Winners have to match 3, 4, 5 or all 6 digits of the winning number in the correct place in the sequence. The lucky winners are sent their prizes automatically each Friday. So if you win a prize you’ll automatically receive it! Play Now What Are My Chances Unity offers the best odds of any platform of its kind. Because it’s a number match game, the odds are fixed. This means that no matter which charity you’re supporting, or how big their lottery is, every lottery number has an equal chance of winning a prize. 3-digit match = 1 in 69 4-digit match = 1 in 823 5-digit match = 1 in 18,518 6-digit match = 1 in 1,000,000 How does it work? The lottery is a number match game using a random number generator. If your digits are in same positions as the digits in the six-digit winning number sequence, you win a prize 3-digit match = 5 entries into the next draw 4-digit match = £25 5-digit match = £1,000 6-digit match = £25,000 Play Now

Explore the Action for BPAN gallery to see the inspiring photos of families, fundraisers, and moments that bring families and community together in the fight against BPAN. Gallery Action for BPAN would be thrilled to showcase your incredible fundraising efforts in our gallery! We would love to highlight the impact you're making, so please send us your photos and videos to be featured. Your contributions help raise awareness and support for BPAN, and we can't wait to share your inspiring work with our community! Event Galleries AFB Golf Day 2025 VEIW GALLARY Wellness Day 2025 VEIW GALLARY The Marvellous Festival 2025 VEIW GALLARY Step Up For BPAN Snowdon 2025 VEIW GALLARY

Action for BPAN is the first UK charity focused on funding research for a cure or treatment for Beta-propeller Protein-associated Neurodegeneration (BPAN). We are dedicated to making a lasting impact in the lives of families affected by this devastating condition by providing support based on our own lived experience.​ Join us in our mission to fund vital research and raise awareness about BPAN. 1/9 Action for BPAN is the first UK charity focused on funding research for a cure or treatment for Beta-propeller Protein-associated Neurodegeneration (BPAN). We are dedicated to making a lasting impact in the lives of families affected by this devastating condition by providing support based on our own lived experience. Join us in our mission to fund vital research and raise awareness about BPAN. Support Action For BPAN Gallery About Us Action for BPAN was founded in 2024 by two mothers, Kelly Sayers and Nicole Harper, whose daughters have both been diagnosed with BPAN. Our mission is to bring hope to families by supporting them, raising awareness and generating funds for BPAN research. Thanks to the support of our growing community and volunteers, the UK is joining the global effort to find a treatment or cure. Driven by our love and dedication to our incredible daughters, and with your support, we can make a meaningful difference in the lives of those affected by BPAN. Meet The Team Meet Isabella Isabella is our joyful, vibrant 6-year-old daughter, who was diagnosed with BPAN at the age of 3. Her future is uncertain - she may become part of a wonderful story where research leads to a cure or treatment in time, or a heart breaking story of a little girl that has to suffer BPAN's degenerative symptoms like Alzheimer's, Parkinson's, and ultimately, the loss of her movement and communication skills. Our mission is to ensure that Isabella and others like her can live a happy, healthy life, and to prevent future generations from having to endure a life with BPAN. Read Isabella's Story Meet Emily Emily is the youngest of our two incredible children, born on 4th April 2016, she was just 4 years old when we received her BPAN diagnosis. Emily is a very special little girl who leaves a lasting impression on everyone she meets, she is full of love, laughter and happiness. Emily gives the best hugs and has a way, even with her limited communication, of charming you into getting exactly what she wants! Whether that's another play fight with her Daddy or a 3rd and 4th story out of Mummy at bedtime! Read Emily's Story Isabella and Emily are just two of the many amazing children living with BPAN Discover more of the inspiring family stories that make our community so special Family Stories What is BPAN Beta-propeller protein-associated neurodegeneration (BPAN ) is a rare and devastating genetic disorder caused by mutations in the WDR45 gene. This condition leads to the accumulation of iron in the brain , resulting in progressive neurodegeneration . The symptoms experienced by affected children resemble those of Alzheimer's disease , Parkinson's disease , Seizures and dystonia , ultimately leading to a loss of motor and communication function's . Time of degeneration varies but typically begins during adolescence, a time when most children are moving forward into the world. Unfortunately, for children with BPAN, this is when they begin to lose the skills they have worked so hard to develop. Learn More LATEST POSTS 1 2 3 4 WHAT WE DO Our Impact We understand the immense challenges faced by families affected by BPAN, and at Action for BPAN, we are dedicated to creating a meaningful impact. Through the funds we raise, we support vital BPAN research, drive awareness initiatives, and organize diverse fundraising efforts, including community events, sponsored challenges, corporate partnerships, and online campaigns. Beyond funding research, we are deeply committed to being there for affected families, offering support, understanding, and guidance through our own lived experiences. Our ultimate goal is to accelerate the development of effective treatments or a cure while providing hope and a strong sense of community for those navigating this journey. Research At Action for BPAN, we are committed to ensuring our community is kept informed about the progress of BPAN research as much as possible. We understand how isolating and frustrating it can be to feel uninformed, especially when the stakes are so high for families affected by this devastating condition. That’s why transparency and communication are at the heart of what we do. We will share regular updates through our website and social media channels, providing the latest news on research breakthroughs and milestones achieved. Whenever possible, we’ll collaborate directly with researchers to relay accurate and accessible information. Our goal is to ensure that no family feels uninformed or unsupported in this journey. Research Updates Fundraising Looking to make a difference? Amazing! Join our fundraising efforts and help us come up with new and creative ideas to support Action for BPAN. Your involvement can have a profound impact on the lives of those affected by this devastating condition. Together, we can accelerate the pace of research and bring hope to families who need it most. Fundraising is not only crucial but also an incredibly rewarding and positive experience. If you’re unsure where to start, we’re here to guide and coach you every step of the way, helping you turn your passion into action. Whether it’s organizing a community event, taking on a personal challenge, or finding new ways to raise awareness, you’ll be part of a movement that truly makes a difference. Join us and see how empowering it can be to help change lives while building connections and spreading hope. Help us Fundraise Volunteering Are you passionate about making a difference? Join us as a volunteer and use your unique skills and experience to support our mission. We welcome individuals who share our dedication to creating meaningful change in the BPAN community and are committed to making a positive impact. Whether you can offer your time, expertise, or creative ideas, your contribution will play a vital role in driving our work forward. Become a part of something truly special and help us bring hope to families affected by BPAN. Together, we can make a lasting difference. Apply to Volunteer