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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Title: 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.
Authors: Knight EMP; Epilepsy Center, Cleveland Neurological Institute, Cleveland Clinic, Cleveland, OH, USA. Electronic address: pestane@ccf.org., Amin S; Pediatric Neurology, University Hospitals Bristol and Weston, Bristol, UK., Bahi-Buisson N; Pediatric Neurology, Necker Enfants Malades University Hospital, Paris, France., Benke TA; Department of Pediatrics and Neurology, University of Colorado School of Medicine, Children's Hospital Colorado, Aurora, CO, USA., Cross JH; UCL NIHR BRC Great Ormond Street Institute of Child Health, London, UK., Demarest ST; Department of Pediatrics and Neurology, University of Colorado School of Medicine, Children's Hospital Colorado, Aurora, CO, USA., Olson HE; Division of Epilepsy and Clinical Neurophysiology and Epilepsy Genetics Program, Department of Neurology, Boston Children's Hospital and Harvard Medical School, Boston, MA, USA., Specchio N; Rare and Complex Epilepsy Unit, Department of Neuroscience, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Fleming TR; Department of Biostatistics, University of Washington, Seattle, WA, USA., Aimetti AA; Marinus Pharmaceuticals, Radnor, PA, USA., Gasior M; Marinus Pharmaceuticals, Radnor, PA, USA., Devinsky O; New York University Langone Comprehensive Epilepsy Center, New York, NY, USA.
Corporate Authors: Marigold Trial Group
Source: The Lancet. Neurology [Lancet Neurol] 2022 May; Vol. 21 (5), pp. 417-427.
Publication Type: Clinical Trial, Phase III; Journal Article; Randomized Controlled Trial
Journal Info: Publisher: Lancet Pub. Group Country of Publication: England NLM ID: 101139309 Publication Model: Print Cited Medium: Internet ISSN: 1474-4465 (Electronic) Linking ISSN: 14744422 NLM ISO Abbreviation: Lancet Neurol Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1474-4465
DOI:10.1016/S1474-4422(22)00077-1