Rapalogues for Autism Phenotype in TSC: A Feasibility Study

Principal Investigator: Tanjala Gipson

Tuberous sclerosis complex (TSC) is associated with autism and seizures. Recent studies in animals using rapamycin have shown cognitive improvement. Human studies have demonstrated a reduction in tumor burden, subjective cognitive improvement, and decreased seizure frequency. The goal of this study is to assess the feasibility and safety of administering sirolimus for self-injury in participants with TSC.