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Huntington's Disease Clinical Trial At USF Explores Gene Silencing

Aug 8, 2019

A phase III clinical trial is underway at the University of South Florida for a drug that targets the genetic cause of Huntington’s Disease, or HD, which is described as having a mix of Parkinson’s, Lou Gehrig’s disease, and Alzheimer’s.

Health New's Florida's Daylina Miller talked with USF Health neurologist Dr. Juan Sanchez-Ramos about what that means for a disease that has a 50%  chance of being passed down to children.

Despite more than 25 years of research, advances in understanding disease biology remains limited and there are currently no therapies that can prevent, slow or stop HD progression.

This clinical trial, the final step before heading to the U.S. Food and Drug Administration for a lengthy approval process, is testing what’s being hailed as the "most advanced HD therapy in development, RG6042.” Recent research shows this gene-silencing treatment, which is being administered through a spinal tap in the trial, reduced the amount of the illness-inducing protein in a study.

The enrollment period for the clinical trials is over.