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Long-term treatment with ganaxolone for seizures associated with cyclin-dependent kinase-like 5 deficiency disorder: Two-year open-label extension follow-up.

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第一作者： Heather E,Olson

第一单位： Department of Neurology, Division of Epilepsy and Clinical Neurophysiology, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts, USA.

作者： Heather E,Olson ^[1] ; Sam,Amin ^[2] ; Nadia,Bahi-Buisson ^[3] ; Orrin,Devinsky ^[4] ; Eric D,Marsh ^[5] ; Elia,Pestana-Knight ^[6] ; Rajsekar R,Rajaraman ^[7] ; Alex A,Aimetti ^[8] ; Eva,Rybak ^[8] ; Fanhui,Kong ^[8] ; Ian,Miller ^[8] ; Joseph,Hulihan ^[8] ; Scott,Demarest ^[9]

作者单位： Department of Neurology, Division of Epilepsy and Clinical Neurophysiology, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts, USA. ^[1] University Hospitals Bristol and Weston, Bristol, UK. ^[2] Pediatric Neurology, Necker Enfants Malades University Hospital, Paris, France. ^[3] New York University Langone Comprehensive Epilepsy Center, New York, New York, USA. ^[4] Departments of Neurology and Pediatrics, University of Pennsylvania Perelman School of Medicine and Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA. ^[5] Epilepsy Center, Cleveland Clinic, Cleveland, Ohio, USA. ^[6] UCLA Mattel Children's Hospital, Los Angeles, California, USA. ^[7] Marinus Pharmaceuticals, Inc., Radnor, Pennsylvania, USA. ^[8] Department of Pediatrics and Neurology, University of Colorado School of Medicine, Precision Medicine Institute, Children's Hospital Colorado, Aurora, Colorado, USA. ^[9]

关键词 Clinical Global Impression-Improvement open-label extension phase 3