Everolimus precision therapy for the GATOR1-related epilepsies: a case series.
Patrick B MoloneyHugh KearneyKatherine A BensonDaniel J CostelloGianpiero L CavalleriKathleen M GormanBryan J LynchNorman DelantyPublished in: European journal of neurology (2023)
Our study provides the first human data on the potential benefit of everolimus precision therapy for epilepsy caused by DEPDC5 loss-of-function variants. Further studies are needed to support our findings.