Unravel Biosciences, Inc.

This early study tests the safety and effects of vorinostat, a drug currently used for cancer, in 5 people with Pitt Hopkins syndrome (ages 3-21). Each person will try a placebo and two different doses of the drug to see if it helps with symptoms and is safe. The goal is to gathe…

This early-stage study tests the safety and effectiveness of vorinostat in 15 girls and young women (ages 6-21) with Rett syndrome. Each participant acts as their own control, receiving placebo first, then two different doses of the drug. The goal is to see if vorinostat can impr…