Prion Disease Clinical Trial NN112

Dr. Gregg Day and Drs. Sonia Vallabh and Eric Minikel discuss scientific insights and the future of prion disease treatment, highlighting the importance of early diagnosis, personalized medicine, and hope for affected families.  Learn about the clinical trial.   Learn more about the Prion Alliance.  Show transcript:  Dr. Gregg Day: This is Gregg Day with Neurology Minute. I've just been speaking with Eric Minikel and Sonia Vallabh, a husband and wife team at the heart of the PRiSM trial, a first-in-human study of a prion protein-lowering, divalent, small-interfering RNA for patients with symptomatic prion disease. Eric and Sonia, could you provide us with a brief overview of the PRiSM trial and what this first-in-human study seeks to accomplish? Dr. Eric Minikel: The PRiSM trial is testing a short interfering RNA designed to bind the RNA that encodes the prion protein. That is the protein that causes Prion disease. We are at the heart of what causes this disease. Through doing this, we hope to make prion disease a treatable and preventable condition. We both want to stabilize symptomatic patients and prevent the disease in people who are at genetic risk. This is a personal mission for us. Sonia is a carrier of a prion disease mutation that she inherited from her mother who died of prion disease, and we, along the way, aspire to be a different kind of sponsor. We want to create our own clinical data that are shareable learnings for the entire field. Dr. Gregg Day: This is Gregg Day with Neurology Minute. Thanks for listening.