Results 81 to 90 of about 86,962 (305)

Severity of effect considerations regarding the use of mutation as a toxicological endpoint for risk assessment: A report from the 8th International Workshop on Genotoxicity Testing (IWGT)

Environmental and Molecular Mutagenesis, EarlyView.
Abstract Exposure levels without appreciable human health risk may be determined by dividing a point of departure on a dose–response curve (e.g., benchmark dose) by a composite adjustment factor (AF). An “effect severity” AF (ESAF) is employed in some regulatory contexts.
Barbara L. Parsons, Marc A. Beal, Kerry L. Dearfield, George R. Douglas, Min Gi, B. Bhaskar Gollapudi, Robert H. Heflich, Katsuyoshi Horibata, Michelle Kenyon, Alexandra S. Long, David P. Lovell, Anthony M. Lynch, Meagan B. Myers, Stefan Pfuhler, Alisa Vespa, Andreas Zeller, George E. Johnson, Paul A. White   +17 more
wiley   +1 more source

Duchenne muscular dystrophy [PDF]

Biochemical Society Transactions, 1984
What is Duchenne muscular dystrophy? Duchenne muscular dystrophy is a muscle-wasting condition caused by the lack of a protein called dystrophin. It usually affects only boys. About 100 boys with Duchenne muscular dystrophy are born in the UK each year and there are about 2,500 boys and young men known to be living with the condition in the UK at any ...
openaire   +6 more sources

Prednisone in Duchenne dystrophy

Neuromuscular Disorders, 1991
The results of a randomized controlled trial of daily Prednisone conducted in 99 boys aged five to 15 years with Duchenne dystrophy are reported from the Department of Neurology, University of Rochester, NY and five collaborating institutions.
openaire   +7 more sources

Hope and the Life Course: Results From a Longitudinal Study of 25,000 Adults

Health Economics, EarlyView.
ABSTRACT This paper reports the first large‐scale longitudinal links between one of the least known dimensions of wellbeing—hope—and long‐term outcomes in a range of life arenas. Hope has agentic properties which are relevant to people's future outcomes. Following 25,000 randomly sampled Australian adults over a period of 14 years from 2007 to 2021 (N >
Carol Graham, Redzo Mujcic
wiley   +1 more source

Development of a Clinical Global Impression of Change (CGI-C) and a Caregiver Global Impression of Change (CaGI-C) measure for ambulant individuals with Duchenne muscular dystrophy

Health and Quality of Life Outcomes, 2021
Background In clinical trials for rare diseases, such as Duchenne muscular dystrophy, clinical outcome assessments (COA) used to assess treatment benefit are often generic and may not be sensitive enough to detect change in specific patient populations ...
Hannah Staunton, Claire Trennery, Rob Arbuckle, Maitea Guridi, Elena Zhuravleva, Pat Furlong, Ryan Fischer, Rebecca Hall   +7 more
doaj   +1 more source

New insights into applications of base editor in hereditary disorders

Interdisciplinary Medicine, EarlyView.
Abstract Hereditary disorders are a group of diseases caused by genetic mutations or chromosomal variations. Although the incidence of each genetic disorder is relatively low, patients affected by the disease generally experience a range of severe symptoms, including blindness, disability, and even premature death. In addition, the available treatments
Maoping Cai, Linhui Zhang, Liqing Li, Yue Liu, Canbin Lv, Yan Shi, Jianyuan Zhao, Dingwei Ye, Yuanyuan Qu   +8 more
wiley   +1 more source

Evolution of therapeutic management of patients with ANCA associated vasculitis in France after licensing Rituximab use

BMC Rheumatology
Introduction In 2013, rituximab was approved in France for the treatment of ANCA-associated vasculitis (AAV). The aim of the study was to compare the treatment and health events of adult incident patients with granulomatosis with polyangiitis (GPA) and ...
Cécile-Audrey Durel, Eric Simon Thervet, Dominique Chauveau, Aurélie Schmidt, Benjamin Terrier, Pierre M Bataille   +5 more
doaj   +1 more source

Being ambulatory does not secure respiratory functions of Duchenne patients

Annals of Indian Academy of Neurology, 2011
Aim: The aim of this work was to assess the respiratory functions of ambulatory Duchenne patients and to propose an earlier time period for intervention. Materials and Methods: Lung functions and North Star Ambulatory Assessment (NSAA) scores of Duchenne
Baris Ekici, Yakup Ergül, Burak Tatli, Feride Bilir, Fatih Binboga, Ayse Süleyman, Zeynep Tamay, Mine Çaliskan, Nermin Güler   +8 more
doaj   +1 more source

Illuminating Satellite Cells: Light Sheet Fluorescence Microscopy for 3D Imaging of Murine Skeletal Muscles Damaged by Ex Vivo Forced Eccentric Contraction

Microscopy Research and Technique, EarlyView.
This study presents the light sheet fluorescence microscopy (LSFM) as a tool for 3D‐imaging of whole skeletal muscle to reveal satellite cells (SCs), the muscle stem cells that activate following damage to repair injured tissue. After tissue clearing and whole‐mount staining process aimed to enable optical access and specific cell labeling (1), murine ...
Rachele Garella, Elisa Imbimbo, Francesco Palmieri, Alessia Tani, Martina Parigi, Flaminia Chellini, Alessandra La Contana, Monica Mattioli Belmonte, Aurora Longhin, Ludovico Silvestri, Chiara Sassoli, Roberta Squecco   +11 more
wiley   +1 more source

Coordinated actions of microRNAs with other epigenetic factors regulate skeletal muscle development and adaptation [PDF]

, 2017
Epigenetics plays a pivotal role in regulating gene expression in development, in response to cellular stress or in disease states, in virtually all cell types.
Adamo, Sergio, Bianchi, Marzia, Moresi, Viviana, Renzini, Alessandra   +3 more
core   +1 more source