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The Latest Data from Lenadogene Nolparvovec Gene Therapy Trials for Leber Hereditary Optic Neuropathy

Author:Newman, Nancy   Yu-Wai-Man, Patrick   Carelli, Valerio   Biousse, Valerie   Taiel, Magali   Sahel, José-Alain   

Session Name:S14: Neuro-ophthalmology/Neuro-otology  

Topic:Neuro-ophthalmology/Neuro-otology  

Program Number:S14.001  

Author Institution:Emory University School of Medicine, Atlanta, GA  University of Cambridge, Newcastle Upon Tyne, United Kingdom  University of Bologna, Bologna, Italy  GenSight Biologics, Paris, France  

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ReNeu: A Pivotal Phase 2b Trial of Mirdametinib in Children and Adults With Neurofibromatosis Type 1 (NF1)-Associated Symptomatic Plexiform Neurofibroma (PN)

Author:Shuhaiber, Hans   Moertel, Christopher   Hirbe, Angela   Viskochil, David   Sidhu, Alpa   Bielamowicz, Kevin   Weber, Michael   Lokku, Armend   Smith, L. Mary   Weintraub, Lauren   McNall-Knapp, Rene   Hajjar, Fouad   Foreman, Nicholas   Gershon, Timothy   Babovic-Vuksanovic, Dusica   

Session Name:S29: Neuro-oncology  

Topic:Neuro-oncology  

Program Number:S29.003  

Author Institution:University of Florida Clinical Research Center, Gainesville, FL  University of Minnesota, Minneapolis, MN  Washington University School of Medicine, St. Louis, MO  University of Utah, Salt Lake City, UT  University of Iowa Hospitals and Clinics, Iowa City, IA  The University of Arkansas for Medical Sciences/Arkansas Children's Hospital, Little Rock, AR  SpringWorks Therapeutics, Inc, Stamford, CT  Formerly SpringWorks Therapeutics, Inc, Stamford, CT  Albany Medical Center, Albany, NY  University of Oklahoma Health Sciences Center, Oklahoma City, OK  AdventHealth for Children, Orlando, FL  Children’s Hospital Colorado, Aurora, CO  Emory University School of Medicine, Atlanta, GA  Mayo Clinic, Rochester, MN  

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Vertebrobasilar Dolichoectasia Causing Ischemic and Hemorrhagic Stroke

Author:Gao, Xiaoyi   Jillella, Dinesh   

Session Name:P5: Cerebrovascular Disease and Interventional Neurology: Non-inflammatory Vasculopathies  

Topic:Cerebrovascular Disease and Interventional Neurology  

Program Number:P5.014  

Author Institution:Emory University School of Medicine, Atlanta, GA  Emory University School of Medicine / Grady Memorial Hospital, Atlanta, GA  

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Post-hoc Analysis of the LEROS Study: Impact of Time to Idebenone Initiation on Visual Acuity in Leber Hereditary Optic Neuropathy

Author:Newman, Nancy J   Yu-Wai-Man, Patrick   Carelli, Valerio   Llòria, Xavier   Silva, Magda Joana   Klopstock, Thomas   

Session Name:P5: Neuro-ophthalmology/Neuro-otology: Clinical Research in Neuro-ophthalmology  

Topic:Neuro-ophthalmology/Neuro-otology  

Program Number:P5.024  

Author Institution:Emory University School of Medicine, Atlanta, GA  University of Cambridge, Cambridge, United Kingdom  IRCCS Istituto di Scienze Neurologiche di Bologna, Bologna, Italy  Chiesi Farmaceutici S.p.A, Parma, Italy  All is Data, Basel, Switzerland  Friedrich Baur Institute, Munich, Germany  

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Ocular Post-mortem Analyses with Histopathological and Molecular Assessments in Leber Hereditary Optic Neuropathy Following AAV2 Gene Therapy

Author:Biousse, Valérie   Carelli, Valerio   Newman, Nancy   Caporali, Leonardo   Ross-Cisneros, Fred   Boschetti, Elisa   DuBois, Lindreth   Liu, Henry   Ancian, Philippe   Taiel, Magali   Sadun, Alfredo   

Session Name:P5: Neuro-ophthalmology/Neuro-otology: Clinical Research in Neuro-ophthalmology  

Topic:Neuro-ophthalmology/Neuro-otology  

Program Number:P5.025  

Author Institution:University of Bologna, Bologna, Italy  Emory University School of Medicine, Atlanta, GA  GenSight Biologics, Paris, France  Doheny Eye Institute, Los Angeles, CA  

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