Patient Resources

About Tuberous Sclerosis Complex (TSC) and Overactivation of mTORC1

TSC is a genetic disorder frequently associated with medically intractable epilepsy, resulting from selective overactivation of mTOR complex 1 (mTORC1) caused by mutations in the TSC1 or TSC2 genes. Thus, directly targeting the mTORC1 overactivation resulting from TSC mutations with a selective mTORC1 inhibitor is believed to be a highly appropriate therapeutic target to address the significant unmet medical needs of patients with TSC refractory epilepsy. For more information about TSC, please visit the TSC Alliance.

Patient Advocacy Groups

Please visit our Patient Advocacy Group page or click the image below to access information on TSC patient advocacy groups, such as TSC Alliance, TS Alliance and the TSA.