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Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A ND4 Mutation
Nancy J. Newman, Patrick Yu-Wai-Man, Valerio Carelli, Valerie Biousse, Mark L. Moster, Catherine Vignal-Clermont, Robert C. Sergott, Thomas Klopstock, Alfredo A. Sadun, Jean-François Girmens, Chiara La Morgia, Adam A. DeBusk, Neringa Jurkute, Claudia Priglinger, Rustum Karanjia, Constant Josse, Julie Salzmann, François Montestruc, Michel Roux, Magali Taiel, José-Alain Sahel
Jun 07, 2021
Objective: This work aimed to compare the evolution of visual outcomes in Leber hereditary optic neuropathy (LHON) patients treated with intravitreal gene therapy to the spontaneous evolution in prior natural history (NH)...
Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A ND4 Mutation
Nancy J Newman, Patrick Yu-Wai-Man, Valerio Carelli, Valerie Biousse, Mark L Moster, Catherine Vignal-Clermont, Robert C Sergott, Thomas Klopstock, Alfredo A Sadun, Jean-François Girmens, Chiara La Morgia, Adam A DeBusk, Neringa Jurkute, Claudia Priglinger, Rustum Karanjia, Constant Josse, Julie Salzmann, François Montestruc, Michel Roux, Magali Taiel, José-Alain Sahel
Jun 28, 2021
Objective: This work aimed to compare the evolution of visual outcomes in Leber hereditary optic neuropathy (LHON) patients treated with intravitreal gene therapy to the spontaneous evolution in prior natural history (NH)...
Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset.
Nancy J Newman, Patrick Yu-Wai-Man, Valerio Carelli, Mark L Moster, Valerie Biousse, Catherine Vignal-Clermont, Robert C Sergott, Thomas Klopstock, Alfredo A Sadun, Piero Barboni, Adam A DeBusk, Jean François Girmens, Günther Rudolph, Rustum Karanjia, Magali Taiel, Laure Blouin, Gerard Smits, Barrett Katz, José-Alain Sahel, LHON Study Group
Apr 15, 2021
PURPOSE: To evaluate the efficacy of a single intravitreal injection of rAAV2/2-ND4 in subjects with visual loss from Leber hereditary optic neuropathy (LHON). DESIGN: RESCUE is a multicenter, randomized, double-masked...
Humphrey visual field perimetry Leber hereditary optic neuropathy Phase 3 randomized double-masked clinical trial Quality of life best-corrected visual acuity bilateral visual improvement Contrast Sensitivity EFFICACY intravitreal gene therapy retinal anatomic measures Safety Adolescent Adult Aged DNA Mitochondrial Dependovirus Double-Blind Method Electroretinography Female Follow-Up Studies Genetic Therapy Genetic Vectors Humans Intravitreal Injections Male Middle Aged Mutation Optic Atrophy Hereditary Leber Quality of life Time Factors Treatment Outcome Visual Acuity Visual Field Tests Visual Fields Young Adult
Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A <i>ND4</i> Mutation
Nancy J Newman, Patrick Yu-Wai-Man, Valerio Carelli, Valerie Biousse, Mark L Moster, Catherine Vignal-Clermont, Robert C Sergott, Thomas Klopstock, Alfredo A Sadun, Jean-François Girmens, Chiara La Morgia, Adam A DeBusk, Neringa Jurkute, Claudia Priglinger, Rustum Karanjia, Constant Josse, Julie Salzmann, François Montestruc, Michel Roux, Magali Taiel, José-Alain Sahel
Jul 28, 2021
Objective: This work aimed to compare the evolution of visual outcomes in Leber hereditary optic neuropathy (LHON) patients treated with intravitreal gene therapy to the spontaneous evolution in prior natural history (NH)...
Published by: Frontiers in neurology
A simple approach to ranking differentially expressed gene expression time courses through Gaussian process regression.
BACKGROUND: The analysis of gene expression from time series underpins many biological studies. Two basic forms of analysis recur for data of this type: removing inactive (quiet) genes from the study and determining which genes...
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.
Valerio Carelli, Nancy J Newman, Patrick Yu-Wai-Man, Valerie Biousse, Mark L Moster, Prem S Subramanian, Catherine Vignal-Clermont, An-Guor Wang, Sean P Donahue, Bart P Leroy, Robert C Sergott, Thomas Klopstock, Alfredo A Sadun, Gema Rebolleda Fernández, Bart K Chwalisz, Rudrani Banik, Jean François Girmens, Chiara La Morgia, Adam A DeBusk, Neringa Jurkute, Claudia Priglinger, Rustum Karanjia, Constant Josse, Julie Salzmann, François Montestruc, Michel Roux, Magali Taiel, José-Alain Sahel, the LHON Study Group
Jan 12, 2023
Natural history of patients with Leber hereditary optic neuropathy-results from the REALITY study.
Patrick Yu-Wai-Man, Nancy J Newman, Valerio Carelli, Chiara La Morgia, Valérie Biousse, Francesco M Bandello, Catherine Vignal Clermont, Lorena Castillo Campillo, Stephanie Leruez, Mark L Moster, Dean M Cestari, Rod Foroozan, Alfredo Sadun, Rustum Karanjia, Neringa Jurkute, Laure Blouin, Magali Taiel, José-Alain Sahel, LHON REALITY Study Group
Mar 31, 2022
High-flow nasal oxygen vs. standard oxygen therapy for patients undergoing transcatheter aortic valve replacement with conscious sedation
S Scheuermann, A Tan, P Govender, M Mckie, J Pack, G Martinez, F Falter, S George, A A. Klein
Apr 14, 2023
Concrete upgrade to improve the vibration response of timber floors
Jonathan Skinner, Carlos Martins, Julie Bregula, R Harris, K A Paine, P J Walker, Alfredo Dias
Sep 01, 2014
Timber floors suffering from poor serviceability performance can be upgraded with a concrete topping to form a timber–concrete composite. The upgrade stiffens the floor, reducing the perception of a vibration response to dynamic...
Enabling Interoperability in the Internet of Things
Semantic Web technologies act as an interoperability glue among different formats, protocols and platforms, providing a uniform vision of heterogeneous devices and services in the Internet of Things (IoT). Semantic Web...
Published by: IGI Global Scientific Publishing
Achieving robustness to aleatoric uncertainty with heteroscedastic Bayesian optimisation
Ryan-Rhys Griffiths, Alexander A Aldrick, Miguel Garcia-Ortegon, Vidhi Lalchand, Alpha A Lee
Nov 23, 2021
Abstract: Bayesian optimisation is a sample-efficient search methodology that holds great promise for accelerating drug and materials discovery programs. A frequently-overlooked modelling consideration in Bayesian optimisation...
Concrete upgrade to improve the vibration response of timber floors
Jonathan Skinner, Carlos Martins, Julie Bregula, R Harris, K A Paine, P J Walker, Alfredo Dias
Sep 01, 2014
Timber floors suffering from poor serviceability performance can be upgraded with a concrete topping to form a timber–concrete composite. The upgrade stiffens the floor, reducing the perception of a vibration response to dynamic...
The spread of agriculture in Iberia through Approximate Bayesian Computation and Neolithic projectile tools.
In the present article we use geometric microliths (a specific type of arrowhead) and Approximate Bayesian Computation (ABC) in order to evaluate possible origin points and expansion routes for the Neolithic in the Iberian...
A generalized approach for NMR studies of lipid-protein interactions based on sparse fluorination of acyl chains.
Alfredo De Biasio, Alain Ibáñez de Opakua, Mark J Bostock, Daniel Nietlispach, Tammo Diercks, Francisco J Blanco
Feb 08, 2019
Sparse lipid fluorination enhances the lipids' 1H signal dispersion, enables clean molecular distinction by 19F NMR, and evinces micelle insertion of proteins via fluorine-induced signal shifts. We present a minimal fluorination...
Corporate Political Activity and Bribery in Africa
There is significant research on the outcomes of corporate political activity (hereafter CPA). However, despite a few prior studies acknowledging the negative externalities of political activity, little attention has been paid...
A Model of Cloud Forensic Application With Assurance of Cloud Log
The key concepts of digital forensic investigation in cloud computing are examination and investigation. Cybercriminals target cloud-based web applications due to presence of vulnerabilities. Forensic investigation is a complex...
Published by: IGI Global Scientific Publishing
Fast sequence evolution of Hox and Hox-derived genes in the genus Drosophila.
Depression amongst interns working in a tertiary care teaching Hospital in Maharashtra
Background: Internship is a crucial stage of medical school regarding student stress which can affect thepatient care negatively. Retrieving knowledge about presence of depression is therefore important in itself and iffound...
Published by: IJHS Medical Association
Depression amongst interns working in a tertiary care teaching Hospital in Maharashtra
Background: Internship is a crucial stage of medical school regarding student stress which can affect the patient care negatively. Retrieving knowledge about presence of depression is therefore important in itself and if found...
Published by: IJHS Medical Association
The spread of agriculture in Iberia through Approximate Bayesian Computation and Neolithic projectile tools.
In the present article we use geometric microliths (a specific type of arrowhead) and Approximate Bayesian Computation (ABC) in order to evaluate possible origin points and expansion routes for the Neolithic in the Iberian...
Corporate Political Activity and Bribery in Africa
There is significant research on the outcomes of corporate political activity (hereafter CPA). However, despite a few prior studies acknowledging the negative externalities of political activity, little attention has been paid...
Making sense of medieval mouths
Trent M Trombley, Sabrina C Agarwal, Patrick D Beauchesne, Caroline Goodson, Francesca Candilio, Alfredo Coppa, Mauro Rubini
Apr 05, 2019
OBJECTIVES: Bioarchaeological investigations of sex-based differences in the prevalence of dental pathological lesions, particularly caries, have drawn considerable attention, and out of this work, two dominant models have...
Knowledge and Attitude among High School Students towards HIV / AIDS in Rural areas of Belagavi–A Cross Sectional Study
Asha A. Bellad and Rajesh R Kulkarni
Mar 25, 2015
Introduction Since its emergence in 1981, the HIV/AIDS pandemic, which poses a serious challenge to mankind, without doubt, has become one of the most serious infectious diseases. Many adolescents around the world are sexually...
Published by: IJHS Medical Association
Sentiment Analysis on Movie Reviews Dataset Using Support Vector Machines and Ensemble Learning
The internet makes it easier for people to connect to each other and has become a platform to express ideas and share information with the world. The growth of the internet has indirectly led to the development of social...
Published by: IGI Global Scientific Publishing
Right sided eventration of diaphragm with clinodactyly
A. Sareen and K. Mahajan
Apr 21, 2015
Diaphragmatic hernia can be of two types-absence or deficiency of the diaphragm, or eventration resulting in elevation of a portion of the diaphragm as a result of incomplete muscularization. Complete eventration almost...
Published by: IJHS Medical Association
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