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Safety and efficacy of ganaxolone in patients with CDKL5 deficiency disorder: results from the double-blind phase of a randomised, placebo-controlled, phase 3 trial

Elia Pestana‐Knight, Sam Amin, Nadia Bahi‐Buisson, Tim A. Benke, J. Helen Cross, Scott Demarest, Heather E. Olson, Nicola Specchio, Thomas R. Fleming, Alex A. Aimetti, Maciej Ga̧sior, Orrin Devinsky, Marigold Trial Group

2022UCL Discovery (University College London)130 citationsOpen Access PDF

Topics & Concepts

Refractory (planetary science)MedicinePlaceboEpilepsyClinical trialDouble blindEncephalopathyPediatricsPsychiatryInternal medicinePathologyAlternative medicineAstrobiologyPhysicsGenetics and Neurodevelopmental DisordersEpilepsy research and treatmentAdvanced biosensing and bioanalysis techniques
Safety and efficacy of ganaxolone in patients with CDKL5 deficiency disorder: results from the double-blind phase of a randomised, placebo-controlled, phase 3 trial | Litcius