Unravel Biosciences, Inc.

This early-stage trial tests the safety and effectiveness of vorinostat, a drug already used for cancer, in 15 girls and young women with Rett syndrome. Each participant serves as their own control, taking a placebo first, then low and high doses of the drug. The goal is to see i…

This study tests whether vorinostat, a drug already used for cancer, is safe and helpful for people with Pitt Hopkins syndrome, a rare genetic condition that affects development. Five participants aged 3 to 21 will each receive placebo, a low dose, and a higher dose in a personal…