Cannabidiol is a medicine that is being developed for the treatment of TSC patients who experience inadequately-controlled seizures. It is being developed as an oral medicine to be added to existing treatment options for TSC (‘add-on therapy’). Cannabidiol has a unique chemical structure that makes it prevent or stop seizures and is thought to have cumulative effect. If licensed, cannabidiol will offer an add-on treatment option for patients with TSC who experience inadequately-controlled seizures.
Sarizotan is an oral medicinal product that is being developed for the treatment of respiratory symptoms associated with Rett syndrome. Sarizotan works by binding to serotonin and dopamine receptors. By stimulating serotonin and dopamine receptors, sarizotan replaces the effect of some of the missing serotonin in the brain and spinal cord. This is expected to help restore normal breathing rhythm in patients with Rett syndrome. Currently, there is no cure for Rett syndrome and treatment focuses on managing the symptoms. If licensed, sarizotan could become the first therapy approved for treatment of Rett Syndrome patients.