AI Medical Compendium Journal:
Annals of the Child Neurology Society

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Preventative treatment of tuberous sclerosis complex with sirolimus: Phase I safety and efficacy results.

Annals of the Child Neurology Society
OBJECTIVE: Tuberous sclerosis complex (TSC) results from overactivity of the mechanistic target of rapamycin (mTOR). Sirolimus and everolimus are mTOR inhibitors that treat most facets of TSC but are understudied in infants. We sought to understand t...