J Neuroophthalmol 2021 Jul 23. Epub 2021 Jul 23.
Departments of Neurology and Ophthalmology (MLM, RCS, AAD), Wills Eye Hospital and Thomas Jefferson University, Philadelphia, Pennsylvania; Departments of Ophthalmology (NJN, VB), Neurology and Neurological Surgery, Emory University School of Medicine, Atlanta, Georgia; Cambridge Centre for Brain Repair and MRC Mitochondrial Biology Unit (PY-W-M), Department of Clinical Neurosciences, University of Cambridge, Cambridge, United Kingdom; Cambridge Eye Unit (PY-W-M), Addenbrooke's Hospital, Cambridge University Hospitals, Cambridge, United Kingdom; Moorfields Eye Hospital (PY-W-M), London, United Kingdom; UCL Institute of Ophthalmology (PY-W-M), University College London, London, United Kingdom; IRCCS Istituto Delle Scienze Neurologiche di Bologna (VC, MC), UOC Clinica Neurologica, Bologna, Italy; Unit of Neurology (VC), Department of Biomedical and Neuromotor Sciences (DIBINEM), University of Bologna, Bologna, Italy; Institute of Health Research and Policy (MSB), University of Illinois, Chicago, Chicago, Illinois; Statistics Consultant (GS), GenSight Biologics, CaliforniaDepartment of Neuro Ophthalmology and Emergencies (CV-C, RH), Rothschild Foundation Hospital, Paris, France; Centre Hospitalier National D'Ophtalmologie des Quinze Vingts (CV-C, RH), Paris, France; Department of Neurology (TK), Friedrich-Baur-Institute, University Hospital, LMU Munich, Munich, Germany; German Center for Neurodegenerative Diseases (DZNE) (TK), Munich, Germany; Munich Cluster for Systems Neurology (SyNergy) (TK), Munich, Germany; Doheny Eye Center UCLA (AAS, RK), Department of Ophthalmology David Geffen School of Medicine at UCLA, Doheny Eye Institute, Los Angeles, CaliforniaDepartment of Ophthalmology (SP), University Hospital, LMU Munich, Munich, Germany; Department of Ophthalmology (RK), University of Ottawa Eye Institute, Ottawa Canada; GenSight Biologics (LB, MT), Paris, France; Medical Consultant (BK), GenSight Biologics; Sorbonne Université (JAS), INSERM, CNRS, Institut de La Vision, 75012 Paris, France; Fondation Ophtalmologique A. de Rothschild (JAS), 25-29 Rue Manin, Paris; Department of Ophthalmology (JAS), the University of Pittsburgh School of Medicine, PittsburghCHNO des Quinze-Vingts (JAS), Institut Hospitalo-Universitaire FOReSIGHT, INSERM-DGOS CIC 1423, Paris, France.
Objective: This report presents a cross-sectional analysis of the baseline characteristics of subjects with Leber hereditary optic neuropathy enrolled in the gene therapy trials RESCUE and REVERSE, to illustrate the evolution of visual parameters over the first year after vision loss.
Methods: RESCUE and REVERSE were 2 phase III clinical trials designed to assess the efficacy of rAAV2/2-ND4 gene therapy in ND4-LHON subjects. At enrollment, subjects had vision loss for ≤6 months in RESCUE, and between 6 and 12 months in REVERSE. Read More