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Amifampridine safety and efficacy in spinal muscular atrophy ambulatory patients: a randomized,placebo-controlled,crossover phase 2 trial |
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| Authors: | Bonanno Silvia Giossi Riccardo Zanin Riccardo Porcelli Valentina Iannacone Claudio Baranello Giovanni Ingenito Gary Iyadurai Stanley Stevic Zorica Peric Stojan Maggi Lorenzo |
| Affiliation: | 1.Neuroimmunology and Neuromuscular Disease Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Via Celoria 11, 20133, Milan, Italy ;2.Department of Oncology and Onco-Hematology, Postgraduate School of Clinical Pharmacology and Toxicology, University of Milan, Milan, Italy ;3.Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy ;4.Department of Clinical Research and Innovation, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy ;5.SPARC Consulting, Milan, Italy ;6.Developmental Neuroscience Research and Teaching Department, Faculty of Population Health Sciences, Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK ;7.NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Hospital NHS Foundation Trust, London, UK ;8.Catalyst Pharmaceuticals, Inc., Coral Gables, USA ;9.Johns Hopkins All Children’s Hospital, St Petersburg, FL, 33701, USA ;10.Faculty of Medicine, Neurology Clinic, University Clinical Center of Serbia, University of Belgrade, Dr Subotica 6, 11000, Belgrade, Serbia ; |
| Abstract: | Journal of Neurology - Spinal muscular atrophy (SMA) is an autosomal recessive disease where a deficient amount of SMN protein leads to progressive lower motor neuron degeneration. SMN-enhancing... |
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