Unravel Biosciences, Inc.

This early-stage trial is testing the drug vorinostat in 15 girls and young women with Rett syndrome, a rare genetic disorder that affects brain development. Each participant will receive placebo and two different doses of vorinostat, acting as their own control. The study aims t…

This study is testing whether a drug called vorinostat is safe and helpful for people with Pitt Hopkins syndrome, a rare genetic condition that affects development. Five participants will each receive a placebo and two different doses of the drug in a special 'N-of-1' design, mea…