epilepsy, mTORopathies, focal cortical dysplasia, LEATS research hub

Open, randomized, IInd phase clinical study evaluating the safety and efficacy of rapamycin in the treatment of drug-resistant epilepsy in children with rare and ultra rare diseases of the central nervous system associated with the activation of the mTOR pathway : BraimTOR-NEURO

This study is looking at a medicine called rapamycin for children whose epilepsy is difficult to treat. We want to see how safe the medicine is and if it can reduce seizures in children with rare brain conditions linked to a specific pathway called mTOR.