Baseline Characteristics of LHON Subjects in the RESCUE and REVERSE Phase III Gene Therapy Trials

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Identifier 20180306_nanos_posters_161
Title Baseline Characteristics of LHON Subjects in the RESCUE and REVERSE Phase III Gene Therapy Trials
Creator Mark L. Moster; Catherine Vignal; Nancy Newman; Alfredo Sadun; Patrick Yu-Wai-Man; Valerio Carelli; Thomas Klopstock; Adam Debusk; Robert Sergott; Molly Scannel Bryan; Jose-Alain Sahel; Barrett Katz; Scott Uretsky
Affiliation (MLM) Wills Eye Hospital and Sidney Kimmel Medical College of Thomas Jefferson University, Philadelphia, PA; (VC) Fondation A de Rothschild, Paris, France; (NN) Emory, Atlanta, GA; (AS) Doheny Eye Institute and UCLA, Los Angeles, CA; (PYWM) Wellcome Trust Centre and Moorfields Eye Hospital, London, United Kingdom; (VC) University of Bologna, Bologna, Italy; (TK) Friedrich-Baur-Institute, University Hospital of LMU, Munich, Germany; (AD) Wills Eye Hospital, Philadelphia, PA; (RS) Wills Eye Hospital and Optic Nerve Research Center, Philadelphia, PA; (MSB) University of Illinois, Chicago, IL; (JAS) Sorbonne Universites andUniversity of Pittsburgh, Paris and Pittsburgh, France; (BK) Albert Einstein College of Medicine, New York, NY; (SU) Gensight Biologics, Paris, France
Subject Genetic Disease; Optic Neuropathy
Description RESCUE and REVERSE are Phase‑ III, randomized, multicenter, double‑masked, sham‑ controlled trials of rAAV2/2-ND4, an intravitreally injected gene therapy for treatment of ND4‑ LHON. RESCUE subjects had vision loss for ≤6 months and REVERSE subjects from >6 months up to 1 year.
Date 2018-03
Language eng
Format application/pdf
Type Text
Source 2018 North American Neuro-Ophthalmology Society Annual Meeting
Relation is Part of NANOS 2018: Poster Presentations
Collection Neuro-ophthalmology Virtual Education Library: NOVEL http://NOVEL.utah.edu
Publisher Spencer S. Eccles Health Sciences Library, University of Utah
Holding Institution North American Neuro-Ophthalmology Association. NANOS Executive Office 5841 Cedar Lake Road, Suite 204, Minneapolis, MN 55416
Rights Management Copyright 2018. For further information regarding the rights to this collection, please visit: https://NOVEL.utah.edu/about/copyright
ARK ark:/87278/s65q8w8p
Context URL The NANOS Annual Meeting Neuro-Ophthalmology Collection: https://novel.utah.edu/collection/NAM/toc/
Setname ehsl_novel_nam
ID 1309581
Reference URL https://collections.lib.utah.edu/ark:/87278/s65q8w8p
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