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Long-term Follow-up of m.11778G>A MT-ND4-LHON Patients Treated with Lenadogene Nolparvovec Ocular Gene Therapy: the RESTORE Study

Author:Biousse, Valerie   Newman, Nancy   Yu-Wai-Man, Patrick   Carelli, Valerio   Moster, Mark   Vignal-Clermont, Catherine   Klopstock, Thomas   Sadun, Alfredo   Sergott, Robert   Taiel, Magali   Sahel, Jose-Alain   

Session Name:S12: Neuro-ophthalmology/Neuro-otology  

Topic:Neuro-ophthalmology/Neuro-otology  

Program Number:S12.004  

Author Institution:Emory University School of Medicine, Atlanta, GA  Cambridge Centre for Brain Repair and MRC Mitochondrial Biology Unit, Newcastle Upon Tyne, United Kingdom  University of Bologna, Bologna, Italy  Neuro-Ophthalmology -Wills Eye Hospital, Philadelphia, PA  Foundation Ophtalmologique A De Rothschild, Paris 75019, France  University of Munich, Dept. of Neurology, Munich, Germany  Doheny Eye Institute USC Sch., Los Angeles, CA  Wills Eye Hospital, Philadelphia, PA  GenSight Biologics, Paris, France  Institut de la Vision, Pittsburgh, PA  

Indirect Comparison of Lenadogene Nolparvovec Gene Therapy versus Natural History in m.11778G>A MT-ND4 Leber Hereditary Optic Neuropathy Patients

Author:Newman, Nancy   Carelli, Valerio   Yu-Wai-Man, Patrick   Biousse, Valerie   Moster, Mark   Subramanian, Prem   Vignal-Clermont, Catherine   Wang, An-Guor   Donahue, Sean   Leroy, Bart   Sergott, Robert   Klopstock, Thomas   Sadun, Alfredo   Rebolleda, Gema   Chwalisz, Bart   Banik, Rudrani   Taiel, Magali   Sahel, Jose-Alain   

Session Name:S12: Neuro-ophthalmology/Neuro-otology  

Topic:Neuro-ophthalmology/Neuro-otology  

Program Number:S12.005  

Author Institution:Emory University School of Medicine, Atlanta, GA  University of Bologna, Bologna, Italy  Cambridge Centre for Brain Repair and MRC Mitochondrial Biology Unit, Newcastle Upon Tyne, United Kingdom  Neuro-Ophthalmology -Wills Eye Hospital, Philadelphia, PA  6. Sue Anschutz-Rodgers University of Colorado Eye Center, Aurora, CO  Foundation Ophtalmologique A De Rothschild, Paris 75019, France  Taipei Veterans General Hospital, Taipei, Taiwan  Vanderbilt University and Vanderbilt Eye Institute, Nashville, TN  Ghent University Hospital, Ghent, Belgium  Wills Eye Hospital, Philadelphia, PA  University of Munich, Dept. of Neurology, Munich, Germany  Doheny Eye Institute USC Sch., Los Angeles, CA  Ramon y Cajal hospital, Madrid, Spain  Massachusetts General Hospital, Department of Neurology, Boston, MA  Icahn School of Medicine at Mount Sinai, New York, NY  GenSight Biologics, Paris, France  Institut de la Vision, Pittsburgh, PA