The CATS Foundation at the Externally-Led, Patient-Focused Drug Development (EL-PFDD) Meeting for GM2

Last week, the Cure & Action for Tay-Sachs (CATS) Foundation had the incredible opportunity to participate in the Externally-Led, Patient-Focused Drug Development (EL-PFDD) Meeting for GM2 representing the voices of the Tay-Sachs and Sandhoff community. 

This meeting, organised by the amazing team at the National Tay-Sachs & Allied Diseases Association was a pivotal moment for the Tay-Sachs and Sandhoff community globally to share our perspectives, challenges, and hopes directly with the FDA.

Being able to advocate for our community at such a crucial event underscores the importance of collaborative efforts in advancing treatments and improving the lives of those affected by these devastating diseases. Our Co-founder Daniel Lewi joined many amazing speakers who presented during the event which we all hope not only helps us shed light on the urgent need for effective therapies but also highlights the resilience and strength of our community.

The FDA plays a vital role in the drug development process, and having them hear firsthand the experiences and priorities of individuals and families impacted by Tay-Sachs and Sandhoff is invaluable. By amplifying our voices globally, we are paving the way for greater understanding, empathy, and progress in the pursuit of treatments and ultimately a cure.

Our hope is that by sharing our stories and insights, we can drive forward new treatments quicker, accelerating the path towards better outcomes for patients and families. 

If you were unable to join the event, you can watch it at the Youtube link below.

Together, let’s continue to advocate, raise awareness, and drive meaningful change!