Hello from a very wet and windy San Diego! It’s been a great start to WORLD 2024.
The goal of WORLDSymposium is to provide an interdisciplinary forum to explore and discuss specific areas of interest, research and clinical applicability related to lysosomal diseases. We have been busy presenting research findings and attending sessions to share learning on lysosomal diseases and how we can improve things for our community. There is a great turn out from clinical and patient organisations in UK so it has also been lovely to catch up with our friends from the LSD collaborative.
On Sunday Sophie attended a session: The Patient Voice: Is Anyone Listening? which aimed to explore measures that industry and other researchers have taken to better understand what is most important to patients in terms of clinical trial endpoints, unmet needs and goals of therapies. It included presentations and discussions from industry, patient representatives, patient groups and clinicians. The key message was given by Alan Finglas (MSD Action Foundation and winner of the WORLDSymposium Patient Advocate Leader (PAL) Award 2024) focussed on the family perspective and asked why should industry include patients?
We can help to design better trials
We can help them to understand disease better
We can push back when regulators do not understand
We can help deliver viable treatments
Cara O’Neil (Cure Sanfilippo Foundation) had a clear message on how to guard against bias towards patients, assumptions about quality of life and risk tolerance. This is to engage in meaningful ways, ask the source and replace assumptions with facts.
Monday was a full day of meetings and presentations for the team and great to begin meeting people face to face and engaging in meaningful discussions. There was a useful session on how technology can help us to better engage and connect. There was a clear message from Dan Lewi (CEO of The CATS Foundation) who shared how people's education and ability to access digital information and tools has progressed meaning we need to support them in how to interpret the information, understand it and use it to ask healthcare professionals the right questions.
Tuesday was another full day of meetings and Sophie presented two poster presentations on LAL D and MLD. Bob attended meetings with the International MPS Network (IMPSN) to discuss MPS Awareness Day and raising awareness globally and with Fabry International Network (FIN) board as a newly appointed member, representing our Fabry community. LSD Global also came together for the first time, representing patient organisations from eight different areas in rare LSDs to strengthen collaboration, share knowledge, tackle some of the barriers in the clinical trial environment and to amplify the patient voice within LSDs globally. Bob is currently the acting Chair of this group as they look to build this collaboration further.
With plenty more happening up till Friday this week we look forward to more collaborations and networking. For now, Fiona shares her highlight:
Seeing Tomas Baldwin, research fellow at University College London, getting one of the Young Investigator Awards and to see Alan Finglas receive the PAL Award. Both well deserved and worthy of this recognition.