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Results of a Phase III, Randomized, Placebo-controlled Crossover Trial with N-acetyl-L-leucine for Niemann-Pick Disease Type C

Author:Bremova-Ertl, Tatiana   Fields, Taylor   Factor, Mallory   Martakis, Kyriakos   Strupp, Michael   

Session Name:PL5: Clinical Trials Plenary Session  

Topic:Child Neurology and Developmental Neurology  

Program Number:PL5.001  

Author Institution:University Hospital Inselspital Bern, Bern, Switzerland  IntraBio, London, United Kingdom  Department of Child Neurology, Marburg, Germany  Ludwig Maximilians University, Munich, Germany, Munich, Germany  

GAA-FGF14 Disease: Defining Its Frequency, Molecular Basis, and 4-aminopyridine Response in a Large Cohort of Patients with Downbeat Nystagmus

Author:Pellerin, David   Heindl, Felix   Wilke, Carlo   Danzi, Matt   Traschutz, Andreas   Ashton, Catherine   Dicaire, Marie-Josee   Cuillerier, Alexanne   Del Gobbo, Giulia   Boycott, Kym   Claassen, Jens   Rujescu, Dan   Hartmann, Annette   Zuchner, Stephan   Brais, Bernard   Strupp, Michael   Synofzik, Matthis   

Session Name:P6: Movement Disorders: Ataxias 1  

Topic:Movement Disorders  

Program Number:P6.013  

Author Institution:McGill University, Montreal, QC, Canada  Ludwig-Maximilians University, Munich, Germany  University of Tübingen, Tübingen, Germany  University of Miami School of Medicine, Miami, FL  Royal Perth Hospital, Perth, Australia  Children’s Hospital of Eastern Ontario Research Institute, Ottawa, ON, Canada  University Duisburg-Essen, Essen, Germany  Medical University of Vienna, Dept. Psychiatry and Psychotherapy, Vienna, Austria  Medical University of Vienna, Vienna, Germany  University of Tübingen, Tubingen, Germany