The CATS Foundation at WORLDSymposium 2024 – a review of a great week of science and research

We’re thrilled to share the highlights from our experience at the recent WORLDSymposium, where we learnt about the latest developments in the research into lysosomal storage diseases (LSDs), including Tay-Sachs and Sandhoff!

Attending the event was an empowering journey, as we witnessed firsthand the incredible strides being made in understanding and treating the awful diseases that impact our community. From innovative enzyme replacement strategies to potential small molecule therapies, the presentations illuminated a path of hope for individuals and families affected by Tay-Sachs and Sandhoff diseases. We attended many meetings with various companies and are excited about the progress being made as we search for those elusive treatments – every day we are getting closer. 

The event started early for us as our co-founder Daniel Lewi was invited to speak on a panel about “Patient-engagement in a new digital world” during the first session of the symposium. It was a great opportunity for us to share the experiences we have encountered in this area for the Tay-Sachs and Sandhoff community, and also to hear about where we would like to see change.

We were also proud to contribute to the dialogue by co-authoring three posters highlighting our advocacy initiatives and the importance of patient-centered care. These we’re:

1. “Empowering the GM2 gangliosidosis community: Developing a single clinical toolkit for clinicians, patients and caregivers in the UK” – this poster detailed our recently launched clinical toolkit
2. “Sleep disturbance is an important feature of neurological lysosomal disease: For patients and families” – this poster explores the role and importance sleep has on those impacted by LSDs
3. “Swallowing disturbance is an important feature of neurological lysosomal disease” – this poster explores the role that measuring swallowing plays and how it can potentially be used in clinical studies

It was great that we were able to co-author two of the posters with Aidan Gill and Gisela Linthorst from AZAFAROS B.V., plus our friends in advocacy Diana Jussila from National Tay-Sachs & Allied Diseases Association and Joslyn Arnon Crowe from NATIONAL NIEMANN-PICK DISEASE FOUNDATION. 

Beyond the scientific breakthroughs, what truly stood out to us was the spirit of collaboration among everyone who attended. Engaging in meaningful discussions, forging new partnerships, and exchanging insights reinforced our shared commitment to advocating for improved treatments and support for our community.

Leaving the WORLDSymposium, we’re inspired and energized to continue our advocacy efforts, maximising the voices of those impacted by Tay-Sachs and Sandhoff. Together, we’re driving progress, raising awareness, and fostering a brighter future for all affected by these cruel diseases