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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.

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第一作者： Elia M Pestana,Knight

第一单位： Epilepsy Center, Cleveland Neurological Institute, Cleveland Clinic, Cleveland, OH, USA. Electronic address: pestane@ccf.org.

作者： Elia M Pestana,Knight ^[1] ; Sam,Amin ^[2] ; Nadia,Bahi-Buisson ^[3] ; Tim A,Benke ^[4] ; J Helen,Cross ^[5] ; Scott T,Demarest ^[4] ; Heather E,Olson ^[6] ; Nicola,Specchio ^[7] ; Thomas R,Fleming ^[8] ; Alex A,Aimetti ^[9] ; Maciej,Gasior ^[9] ; Orrin,Devinsky ^[10] ; Marigold Trial Group

作者单位： Epilepsy Center, Cleveland Neurological Institute, Cleveland Clinic, Cleveland, OH, USA. Electronic address: pestane@ccf.org. ^[1] Pediatric Neurology, University Hospitals Bristol and Weston, Bristol, UK. ^[2] Pediatric Neurology, Necker Enfants Malades University Hospital, Paris, France. ^[3] Department of Pediatrics and Neurology, University of Colorado School of Medicine, Children's Hospital Colorado, Aurora, CO, USA. ^[4] UCL NIHR BRC Great Ormond Street Institute of Child Health, London, UK. ^[5] Division of Epilepsy and Clinical Neurophysiology and Epilepsy Genetics Program, Department of Neurology, Boston Children's Hospital and Harvard Medical School, Boston, MA, USA. ^[6] Rare and Complex Epilepsy Unit, Department of Neuroscience, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy. ^[7] Department of Biostatistics, University of Washington, Seattle, WA, USA. ^[8] Marinus Pharmaceuticals, Radnor, PA, USA. ^[9] New York University Langone Comprehensive Epilepsy Center, New York, NY, USA. ^[10]