See all the latest news, blogs and updates from the medical sector.
Condition
Update on UK process for exploring international access to FAYUVI
There is no formal NHS pathway to access FAYUVI, which remains an investigational gene therapy in the UK. This article outlines the process a family could take to explore privately funded access to FAYUVI.
06/10/2026
Blog
Together we can transform lives
Our amazing fundraiser have been super busy this summer. From boxing, skydiving, footy and superheroes, it's time to catch up on the latest fundraising round-up.
23/09/2026
Story
Ready for new beginnings
Take a look at all those smiley faces! Whether it's a fresh start or the return to an everyday routine after the summer break, thank you for sharing your updates with our community.
23/09/2026
News
UK MPS Society welcomes FDA approval of UX111 for MPS IIIA
The UK MPS Society welcomes the news that the US Food and Drug Administration (FDA) has approved UX111 for the treatment of paediatric patients with Mucopolysaccharidosis Type IIIA (MPS IIIA), also known as Sanfilippo syndrome Type A
16/09/2026
Blog
New children's book launches to save children like Leni
Little Leni has the most adorable laugh. She is curious, funny and full of energy! But Leni has a big struggle ahead of her. Every penny from this book, based on Leni's story, will help raise awareness of Childhood Dementia in the UK.
08/09/2026
Condition
SMC final review approves life-changing gene therapy for treating MLD
We are delighted to announce that the SMC has recently undertaken its final review of atidarsagene autotemcel (Libmeldy®) and has given a positive recommendation for its use within NHS Scotland.
06/09/2026
Story
Our summer of '26: proms, day trips, festivals and ice-cream
Maddi's mum Shelly looks back at their fun-filled summer months and all the great memories they have made together with family and friends.
26/08/2026
Blog
A new chapter for Sophie
Since recently stepping into the role of Interim CEO, Sophie Thomas reflects on her journey with the MPS Society so far and explains her commitment for her next chapter with the charity.
26/08/2026
Condition
Fabrazyme supply update for our Fabry community
We are sharing an update about the supply of Fabrazyme. You may already be aware that the FDA has raised concerns following a review of Sanofi’s manufacturing site in Ireland and we understand that uncertainty about treatment can be unsettling.
26/08/2026
News
Regulatory update on RGX-121 for MPS II Hunter
Regenxbio have today issued an announcement about its investigational gene therapy RGX-121 for MPS II Hunter syndrome.
