Following Denali's announcement that the FDA has approved an enzyme replacement therapy for the neurological aspects of MPS II, we recognise that our community may have questions.

Leni's story highlights the devastating reality faced by families affected by childhood dementia, why this needs to be a recognised condition and how newborn screening reforms can help others living with rare conditions.

Emily and Leni were invited to join Jesy for the special Mother's Day event she organised to celebrate mums caring for children with disabilities and rare conditions.

We want to reassure families that there are no immediate changes to EHCPs and there is no action you need to take at this stage.

In January, MPS Society member Archie travelled to Paris to share his story at the BioMarin Conference. Read about his trip and how well the interview was received.

A Christmas fayre, carol concert, Santa fun run and the Big Give, it can only be the latest fundraising round-up!

Our teams from the MPS Society and RDRP were delighted to attend WORLDSymposium 2026 in San Diego. Catch up on the presentation highlights.

At the 2026 WORLDSymposium, Group CEO Bob Stevens was honoured with the PAL Award. In his speech, he emphasized the importance of standing together as a global community and that this work must always be about people not profit or politics.

The MPS Society is aware of an announcement from REGENXBIO regarding its investigational gene therapy programs RGX-111 for MPS I Hurler syndrome) and RGX-121 for MPS II (Hunter syndrome).

Meet our amazing team of runners who are currently training hard to take on the ultimate challenge, the 2026 TCS London Marathon.

We need to act now. Read a summary of the key messages from the Childhood Dementia Scotland event in December 2025.

We are deeply disappointed by the UK NSC’s decision not to add MLD to the newborn screening panel and we will not stop fighting for newborn screening for MLD.

CSAC is the MPS Society's Clinical Scientific Advisory Committee. Its chair Fiona Stewart looks back at an eventful year and talks about the exciting research projects the committee was able to support.

Sam, founder of SabreFit explains the inspiration behind his fitness clothing line and why he is donating his profits to the MPS Society this Christmas.

To celebrate their family business turning 100 years old, three brothers from Nantwich, Cheshire decided to embark on the adventure of a lifetime.

For over 20 years, Snugburys have been building amazing straw sculptures in support of charity. In 2024, a giant 45ft straw Paddington Bear appeared in their field to raise awareness for the MPS Society.

Lianne has chosen the MPS Society as her charity for the year and is currently in the middle of a monthly schedule of challenging fundraising activities. She reflects on her achievements so far and why fundraising is so important to her.

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.

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.

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!

Sophie Thomas shares the latest updates from the Newborn Screening Summit & 6th APAC Educational Forum for Families and Caregivers on MPS in Taiwan.

Mel explains why the Independent Adult Weekend and meeting others face to face is so important, especially since she's transitioned to adult services.

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.

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.

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.

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.

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.

This week, our Chief Executive Bob Stevens and RDRP colleague Bennie Marshall-Andrews have been on a vital whirlwind tour of some of our pharma partners in the USA. Bob shares some important updates from the trip.

Dave has recorded an insightful video to offer an insight into his first ever Fabrazyme infusion.

The MPS Society recognises how vital sibling support is. That’s why we want to spotlight Sibs - a charity offering free, practical resources for schools to help siblings feel seen and supported.

Mountain climbs, golf marathons, a Tough Mudder and so much more! It is jam-packed! Get yourself a cuppa and enjoy the latest fundraising round-up!

Read about Olivia's remarkable journey from diagnosis, the challenging pursuit of treatment to fulfilling her ambition of becoming a teacher.

Regardless of location and specialist teams, clinical guidelines exist to ensure the same high standard of care. Learn more about our involvement in their development and see which conditions have so far been published.

We caught up with Scarlett who has been doing some amazing fundraising on behalf of her friend Luna.

We urge our global rare disease community to stand with us and demand smarter, faster and more ethical clinical trial pathways that serve the people who need them.

For families affected by neuronopathic MPS, every second counts. But too often, children are diagnosed too late to take part in clinical trials or access treatments that might help them. That’s why we are calling for urgent changes.

Peaks, marathons, pantomimes and charity football games all awash in a sea of blue! Yes, it can only be one thing, it's time for your latest fundraising blog!

Our Patient Services team has had another eventful month, working tirelessly across the UK to support families, provide education for professionals and advocate for our community at every level.

What a busy month May has been! The MPS Society team continues to champion your voice across a range of national conversations, collaborations and awareness initiatives.

This MPS Awareness Day we joined the IMPSN campaign in sharing the message It’s About Time which aims to raise awareness of MPS and the things our community need now. So, what is it about time for?

This year’s meeting in Lisbon marked the 20th anniversary of the Fabry International Network (FIN), a time to reflect on two decades of collaboration and progress.

We held an event on MPS Awareness Day to say thank you to our supporters and volunteers for their contribution to the charity's work.

We know that living with a rare condition is tough. Some days are good, but some days aren’t! Our goal is to equip you with a ‘bag of tricks’ that you can pick and choose from when you need an extra pick-me-up.

Running, stepping, awareness days and talks, as well as more pub quizzes! Sit back and enjoy our latest edition of our fundraising blog!

Our community is at the heart of everything we do. Making your voices heard is a key priority for us, and this month has been no exception. Read on to find out which meetings and conferences we attended to represent you.

The first Saturday in April is International Fabry Women's Day and we had a lovely chat with Liz who has had a very recent diagnosis with Fabry.

It's been another busy month in which the MPS Society team actively represented you and educated professionals across the UK. Read more about our contributions to the MLD Conference, LINK Meeting, Family Screening Project and more.

Our teams from MPS Society UK and RDRP are sharing their experience of attending WORLDSymposium in San Diego this February. Read more about the connections they have made and the key takeaways from the event.

If you followed last year’s BBC Children in Need programme, you simply couldn’t have missed Ethan who joined the One Show’s CIN Challenge Squad and starred as The Greatest Showman in the live show. Hear all about his incredible experience.

We are excited to offer two exciting, new opportunities for members to actively engage in shaping our work. These are the Rare Voices Group and the MPS Ambassador Programme. Find out how you can get involved.