The CATS Foundation presented a poster titled “Collaboration between patient advocacy and industry to create a master protocol to investigate the novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosides and Ataxia-telangiectasia” at the WORLDSymposium in 2023. The event, held in Orlando enabled us to share our with Intrabio as they developed their clinical trial.
About WORLDSymposium
WORLDSymposium™ is an annual research conference dedicated to lysosomal diseases. WORLD is an acronym that stands for We’re Organizing Research on Lysosomal Diseases. Since its inception as a small group of passionate researchers in 2002, WORLDSymposium has grown to an international research conference that attracts over 2000 participants from more than 50 countries around the globe. WORLDSymposium is designed for basic, translational and clinical researchers, patient advocacy groups, clinicians, and all others who are interested in learning more about the latest discoveries related to lysosomal diseases and the clinical investigation of these advances. Each year, WORLDSymposium presents the latest information from basic science, translational research, and clinical trials for lysosomal diseases.
Collaboration between patient advocacy and industry to create a master protocol to investigate the novel therapy
Highlights
The poster shows how we worked effectively with other advocacy partners in different conditions.
Key message
Working collaboratively can have huge benefits to advocacy and industry.
How to share
The poster can be shared via the button at the top of this page.
