Precision mTOR targeting aims to widen the window in TSC epilepsy
Selective mTORC1 inhibition emerges as a strategy to improve safety and efficacy in rare epilepsy disorder
Companies are turning to tuberous sclerosis complex epilepsy as a proving ground for selective mTORC1 inhibitors that could improve on standard-of-care Afinitor. The appeal is straightforward: the target is already de-risked, and success in TSC could translate to a wider set of diseases.
Tuberous sclerosis complex (TSC) is a rare developmental disorder caused by mTOR hyperactivation, leading to multisystem issues such as seizures and widespread hamartomas...
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