See all the latest news, blogs and updates from the medical sector.
News
New hope for children with devastating rare genetic disorder, thanks to world-first research in Manchester
The parents of a three-year-old boy born with a devastating, life-limiting genetic condition say they are now excited for his future after he received a revolutionary stem cell gene therapy treatment.
24/11/2025
Blog
Sally and Marianne | Big Give Christmas Challenge 2025
Sally and Marianne run the MPS Society community events throughout the year. We caught up with them to discuss how activities are chosen and organised, why affordability matters, plans for 2026 and what makes our events so special.
24/11/2025
Blog
Together we can transform lives
Great runs in London, Manchester and Scotland. Dancers, carnivals, birthday fundraisers with a sprinkling of Christmas in the air. Here's the latest fundraising round-up!
20/11/2025
News
Lessons from Taiwan
Sophie Thomas shares the latest updates from the Newborn Screening Summit & 6th APAC Educational Forum for Families and Caregivers on MPS in Taiwan.
20/11/2025
Story
Mel's story | Big Give Christmas Challenge 2025
Mel explains why the Independent Adult Weekend and meeting others face to face is so important, especially since she's transitioned to adult services.
18/11/2025
Story
Allison's story | Big Give Christmas Challenge 2025
For Allison, the annual Weekend of Remembrance and Hope each September is a profound source of comfort and connection. The weekend offers families a safe and supportive space to honour their loved ones alongside others who truly understand.
11/11/2025
Blog
World Orphan Drug Congress Europe 2025
The World Orphan Drug Congress (WODC) is dedicated to advancing the development and delivery of treatments for rare diseases. It brings together industry leaders, researchers, policymakers, patient advocates and biotech innovators.
05/11/2025
Story
Emily's story | Big Give Christmas Challenge 2025
Emily shares why community events mean so much to our families, from feeling accepted and understood to enjoying a day out away from the everyday challenges and creating new friendships.
04/11/2025
News
Get involved in research more easily with RDRP's research registration
Help shape the future of rare conditions by signing up to take part in research. Find out why it's important and how to hear about research opportunities.
24/10/2025
News
Bob Stevens selected for prestigious 2026 PAL Award
After reviewing numerous nominations and considering many amazing individuals, the WORLDSymposium 2026 Awards Committee has selected Bob Stevens as the recipient of the 2026 Patient Advocate Leader (PAL) Award.
