This year's International MPS Symposium brought together patients, families, clinicians and researchers from around the world for several days of learning and loads of fun too. Alison and Bethanie from the Patient Services Team were excited to be joined by our ambassadors Sam, Jacob and Helen and delighted to catch up with some of our members who made the trip to Florence too.
Together they reflect on their experience and share some core messages from the presentations they attended.
Jacob
For Jacob the journey to Florence began with a six hour drive from Devon to Gatwick Airport, which he described as the start of a significant personal milestone as this was his first major trip.
I felt really supported as part of a group where I had a safety net of people around me.
A key part of Jacob’s experience was connecting with others in the MPS community, spending time together exploring Florence, admiring its architecture, museums and enjoying delicious meals out.
Jacob added that the experience had a lasting impact on him:
Talking to my friends, I have come across new ways to think of things. The main impact is that I'm encouraged to continue putting myself in situations out of my comfort zone.
Family programme highlights
From genetics and family planning to mental health, quality of life, neurological complications, treatment advances and global access to care, the sessions reflected on the huge variety of experiences across the MPS community.
One of the most powerful sessions focused on lived experience and we were captivated when MPS Society member Archie described how he educated his classmates about MPS and channelled his determination into adaptive sport and entrepreneurship.
Rachel Siew from Malaysia, who has MPS IVA, spoke about overcoming stigma and misunderstanding to build a career and become a patient advocate while Alexia Brandau from Germany shared the journey from her MPS VI diagnosis to becoming an equestrian, teacher and mother of two. Together, these stories reinforced a message that echoed throughout the symposium:
people living with MPS should never be defined by their diagnosis or prognosis.
A particularly meaningful discussion focused on caregivers, many of whom experience chronic sleep deprivation, hypervigilance and concern about medical emergencies which highlighted that
improving quality of life means supporting the whole family, not just the individual with MPS.
The overall message was one of cautious optimism, stressing that earlier diagnosis, better data, equitable access to care, strong patient advocacy and continued research can continue to improve outcomes for people living with MPS worldwide.
Sam
Sam, who played the lead in a production inspired by his time on a children’s ward only a few months ago, found himself in Italy for the very first time.
I had a great time in Florence. I really enjoyed speaking to people from around the world and understanding their personal experiences of with living with MPS.
Throughout the week, Sam spent time with fellow ambassadors, members and families, building new connections within the international MPS community.
I came away with new friendships and a true appreciation for how supportive the MPS community is. It gave me a real sense of belonging.
The opportunity to meet people from different countries, hear their stories and share experiences made the symposium a memorable and rewarding experience for him.
Helen
Setting off to Florence, Helen promised herself to represent our community to the best of her abilities and to share the highlights of the trip upon her return.
It was an experience that was both inspiring and deeply moving, which has left a lasting impression on me.
You can read up on her candid observations and learn more about the people she met in...
Emily
The team were delighted to run into MPS Society members Emily, Angus and their daughter Leni who travelled to the conference with the aim of meeting the clinicians, research teams and pharmaceutical companies behind the potential clinical trials for MPS IIIB. They hoped to share their personal experience of the condition, provide a family perspective and for those teams to meet Leni to better understand the real impact of the treatments and the probable consequences of any delays. Emily reflects:
We made new friends, shared experiences, built closer relationships with the MPS Society and patient associations worldwide and gained a huge amount of knowledge and hope for the future of these conditions.
Scientific programme highlights
Across the week, discussions ranged from advances in gene therapy and newborn screening to emerging approaches for treating brain, skeletal and other hard-to-reach disease manifestations.
In a session focussing on genetics, diagnostics and unmet needs, early treatment was highlighted as critical, illustrated by a case in which a child treated within days of birth showed minimal disease progression compared with an untreated sibling.
A recurring theme was the need for better outcome measures and biomarkers that can capture slow or subtle clinical changes, which may currently be underestimated in trials.
The importance of equitable access to diagnosis and treatment, including a discussion of global registries and international collaboration to reduce disparities, was also emphasised across the sessions.
Dr Fiona Stewart
Fiona attended in her role as Chair of CSAC, contributing to scientific sessions and wider discussions. Together with Group CEO Bob Stevens, she held a number of productive meetings with pharmaceutical companies and was encouraged by the positive news that there will be a trial for Fucosidosis, one of the related conditions for which there is currently no specific therapy.
She also stressed the value of international collaboration when reconnecting with Professor Meenakshi Bhat, who manages MPS patients in Bangalore in a very different healthcare context and faces many different challenges.
It was also fantastic to meet Sam, Jacob and Archie and catch up with Jib. They are four remarkable young men and I really enjoyed spending time in their company.
Bethanie
Bethanie, Head of Support, attended her first conference as part of the MPS Society. She described her time in Florence as both inspiring and rewarding.
I loved hearing people's stories and learning about their amazing achievements. It was incredibly inspiring to see how many people are living life to the fullest and not letting their condition hold them back.
She also valued the opportunity to connect more closely with MPS Society ambassadors and colleagues.
I'm sure the connections I made will be invaluable.
Alison
Alison, Head of Projects and Communities, draws on nearly sixteen years of attending conferences when she reflects on the International MPS conference as a special event.
For Alison, one of the most striking aspects of international meetings is the sense of visibility and belonging:
When you wander around the streets of the city this conference is held, look around and see lots of people who you recognise as being part of the MPS community, I think that is really special.
Throughout the event, Alison was busy attending the family and scientific programmes, reviewing research posters and speaking with pharmaceutical companies. She sees these events as a valuable chance to learn, both from the latest research and from hearing directly about families’ experiences.
I would really encourage anyone who is thinking about attending a conference to put this one on their list for the future.
