Long-term treatment with ganaxolone for seizures associated with cyclin-dependent kinase-like 5 deficiency disorder: 2-year open-label extension follow-up.
Heather E OlsonSam AminNadia Bahi-BuissonOrrin DevinskyEric D MarshElia Pestana-KnightRajsekar R RajaramanAlex A AimettiEva RybakFanhui KongIan MillerJoseph HulihanScott T DemarestPublished in: Epilepsia (2023)
Sustained reductions in MMSF at 2 years in the OLE support the efficacy of ganaxolone in seizures associated with CDD. Safety findings in the OLE were consistent with the double-blind phase.