Tuberous Sclerosis Drug Pipeline Analysis Report

Tuberous sclerosis, caused by mutations in the genes TSC1 and TSC2, affects around 1 in 6,000 to 1 in 10,000 live births. The overall prevalence of this rare genetic disorder is approximately 1 in 20,000. Currently, there is no cure for tuberous sclerosis, and the available treatments are symptomatic and supportive, including early interventions to reduce developmental delays…