Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G>A MT-ND4 Mutation.
Autorzy:
Carelli V; Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Istituto delle Scienze Neurologiche di Bologna, Programma di Neurogenetica, Via Altura, 3, 40139, Bologna, BO, Italy. .; Department of Biomedical and Neuromotor Sciences (DIBINEM), University of Bologna, Bologna, Italy. . Newman NJ; Department of Ophthalmology, Emory University School of Medicine, Atlanta, GA, USA.; Department of Neurology, Emory University School of Medicine, Atlanta, GA, USA.; Department of Neurological Surgery, Emory University School of Medicine, Atlanta, GA, USA. Yu-Wai-Man P; Department of Clinical Neurosciences, Cambridge Centre for Brain Repair and MRC Mitochondrial Biology Unit, University of Cambridge, Cambridge, UK.; Cambridge Eye Unit, Addenbrooke's Hospital, Cambridge University Hospitals, Cambridge, UK.; Moorfields Eye Hospital, NHS Foundation Trust, London, UK.; Institute of Ophthalmology, University College London, London, UK. Biousse V; Department of Ophthalmology, Emory University School of Medicine, Atlanta, GA, USA.; Department of Neurology, Emory University School of Medicine, Atlanta, GA, USA.; Department of Neurological Surgery, Emory University School of Medicine, Atlanta, GA, USA. Moster ML; Department of Neurology, Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA, USA.; Department of Ophthalmology, Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA, USA. Subramanian PS; Sue Anschutz-Rodgers University of Colorado Eye Center, University of Colorado School of Medicine, Aurora, CO, USA. Vignal-Clermont C; Department of Neuro Ophthalmology and Emergencies, Rothschild Foundation Hospital, Paris, France.; Centre d'Investigation Clinique, Centre Hospitalier National d'Ophtalmologie des Quinze Vingts, Paris, France. Wang AG; Department of Ophthalmology, Taipei Veterans General Hospital, National Yang Ming Chiao Tung University, Taipei, Taiwan. Donahue SP; Department of Ophthalmology, Neurology, and Pediatrics, Vanderbilt Eye Institute, Vanderbilt University Medical Center, Vanderbilt University, Nashville, TN, USA. Leroy BP; Department of Ophthalmology and Center for Medical Genetics, Ghent University Hospital, Ghent, Belgium.; Department of Head & Skin, Ghent University, Ghent, Belgium. SergottRC; Department of Neurology, Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA, USA.; Department of Ophthalmology, Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA, USA. Klopstock T; Department of Neurology, Friedrich-Baur-Institute, University Hospital, Ludwig-Maximilians-University Munich, Munich, Germany.; German Center for Neurodegenerative Diseases (DZNE), Munich, Germany.; Munich Cluster for Systems Neurology (SyNergy), Munich, Germany. Sadun AA; David Geffen, Doheny Eye Institute, School of Medicine, University of California, Los Angeles, CA, USA. Rebolleda Fernández G; Department of Ophthalmology, Alcala University, Madrid, Spain. Chwalisz BK; Department of Ophthalmology, Massachusetts Eye & Ear, Harvard Medical School, Boston, MA, USA. Banik R; Department of Ophthalmology, Icahn School of Medicine at Mount Sinai, New York, NY, USA. Girmens JF; Centre d'Investigation Clinique, Centre Hospitalier National d'Ophtalmologie des Quinze Vingts, Paris, France. La Morgia C; Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Istituto delle Scienze Neurologiche di Bologna, Programma di Neurogenetica, Via Altura, 3, 40139, Bologna, BO, Italy.; Department of Biomedical and Neuromotor Sciences (DIBINEM), University of Bologna, Bologna, Italy. DeBusk AA; Department of Neurology, Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA, USA.; Department of Ophthalmology, Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA, USA. Jurkute N; Moorfields Eye Hospital, NHS Foundation Trust, London, UK.; Institute of Ophthalmology, University College London, London, UK.; Department of Neuro-Ophthalmology, The National Hospital for Neurology and Neurosurgery, University College London Hospitals NHS Foundation Trust, London, UK. Priglinger C; Department of Ophthalmology, University Hospital, Ludwig-Maximilians-University Munich, Munich, Germany. Karanjia R; David Geffen, Doheny Eye Institute, School of Medicine, University of California, Los Angeles, CA, USA.; Department of Ophthalmology, University of Ottawa Eye, Ottawa, ON, Canada. Josse C; eXYSTAT, Data Management and Statistic, Malakoff, France. Salzmann J; Medical and Regulatory Consulting, Paris, France. Montestruc F; eXYSTAT, Data Management and Statistic, Malakoff, France. Roux M; GenSight Biologics, Paris, France. Taiel M; GenSight Biologics, Paris, France. Sahel JA; Institut de la Vision, Sorbonne Université, INSERM, CNRS, Paris, France.; Rothschild Foundation Hospital, Paris, France.; Department of Ophthalmology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.; Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts, Institut Hospitalo-Universitaire FOReSIGHT, INSERM-DGOS CIC 1423, Paris, France.
Risdiplam treatment has not led to retinal toxicity in patients with spinal muscular atrophy.
Autorzy:
SergottRC; Department of Neuro-Ophthalmology, Wills Eye Hospital, Philadelphia, USA.; Annesley EyeBrain Center, Thomas Jefferson University, Philadelphia, USA. Amorelli GM; Paediatric Neurology and Nemo Center, Catholic University and Policlinico Gemelli, Rome, Italy. Baranello G; The Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, & Great Ormond Street Hospital Trust, London, UK.; Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy. Barreau E; Institute of Myology, Paris, France. Beres S; Department of Neurology, Department of Ophthalmology, Stanford University, Palo Alto, California, USA. Kane S; Columbia University Medical Center, New York, USA. Mercuri E; Paediatric Neurology and Nemo Center, Catholic University and Policlinico Gemelli, Rome, Italy. Orazi L; Paediatric Neurology and Nemo Center, Catholic University and Policlinico Gemelli, Rome, Italy. SantaMaria M; Annesley EyeBrain Center, Thomas Jefferson University, Philadelphia, USA. Tremolada G; Neuro-Ophthalmology Center, Ophthalmology Department, IRCCS Istituto Auxologico Italiano, Milan, Italy. Santarsiero D; Neuro-Ophthalmology Center, Ophthalmology Department, IRCCS Istituto Auxologico Italiano, Milan, Italy. Waskowska A; Department of Developmental Neurology, Medical University of Gdańsk, Gdańsk, Poland. Yashiro S; Department of Ophthalmology, National Center for Global Health and Medicine (NCGM), Tokyo, Japan. Denk N; Roche Pharma Research and Early Development, Pharmaceutical Sciences, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland. Fürst-Recktenwald S; Pharma Development Neurology, F. Hoffmann-La Roche Ltd, Basel, Switzerland. Gerber M; Pharma Development, Safety, F. Hoffmann-La Roche Ltd, Basel, Switzerland. Gorni K; PDMA Neuroscience and Rare Disease, F. Hoffmann-La Roche Ltd, Basel, Switzerland. Jaber B; Pharma Development, Safety, F. Hoffmann-La Roche Ltd, Basel, Switzerland. Jacobsen B; Roche Pharma Research and Early Development, Pharmaceutical Sciences, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland. Mueller L; Roche Pharma Research and Early Development, Pharmaceutical Sciences, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland. Nave S; Roche Pharma Research and Early Development, Pharmaceutical Sciences, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland. Scalco RS; Pharma Development Neurology, F. Hoffmann-La Roche Ltd, Basel, Switzerland. Marzoli SB; Neuro-Ophthalmology Center, Ophthalmology Department, IRCCS Istituto Auxologico Italiano, Milan, Italy.
Atrial fibrillation post central retinal artery occlusion: Role of implantable loop recorders.
Autorzy:
Watson RA; Division of Cardiology, Department of Medicine, Thomas Jefferson University Hospital, Philadelphia, PA. Wellings J; Division of Cardiology, Department of Medicine, Thomas Jefferson University Hospital, Philadelphia, PA. Hingorani R; Division of Cardiology, Department of Medicine, Thomas Jefferson University Hospital, Philadelphia, PA. Zhan T; Thomas Jefferson University. Frisch DR; Division of Cardiology, Department of Medicine, Thomas Jefferson University Hospital, Philadelphia, PA. Ho RT; Division of Cardiology, Department of Medicine, Thomas Jefferson University Hospital, Philadelphia, PA. Pavri BB; Division of Cardiology, Department of Medicine, Thomas Jefferson University Hospital, Philadelphia, PA. SergottRC; Wills Eye Hospital, The WIlliam Annesley Eye Brain Center, Philadelphia, PA. Greenspon AJ; Division of Cardiology, Department of Medicine, Thomas Jefferson University Hospital, Philadelphia, PA.
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