Pitt-Hopkins Syndrome
Pitt-Hopkins syndrome (PTHS) is an extremely rare disease with approximately 500 confirmed cases worldwide. Individuals suffering from the disease have recognizable facial features and poor intellectual, muscular and psychomotor development. They appear as “excessive” smiling, pleasant and happy personalities. Though most PTHS cases have occurred spontaneously, some exceptions of familial heredity have been reported.
Purposeful lead
After a series of successful preclinical studies, including dose-ranging, Purposeful is ready to take further steps in evaluating the proposed drug candidates. Purposeful’s leading repurposed drug, widely studied for its safe and potent profile, is undergoing further investigation.