Duchenne and Becker muscular dystrophies: a review of animal models, clinical end points, and biomarker quantification

K Wilson, C Faelan, JC Patterson-Kane… - Toxicologic …, 2017 - journals.sagepub.com
Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) are
neuromuscular disorders that primarily affect boys due to an X-linked mutation in the DMD …

[HTML][HTML] Evolving therapeutic strategies for Duchenne muscular dystrophy: targeting downstream events

JG Tidball, M Wehling-Henricks - Pediatric Research, 2004 - nature.com
Duchenne muscular dystrophy (DMD) is a progressive, lethal, muscle wasting disease that
affects 1 of 3500 boys born worldwide. The disease results from mutation of the dystrophin …

[HTML][HTML] Use of capillary Western immunoassay (Wes) for quantification of dystrophin levels in skeletal muscle of healthy controls and individuals with Becker and …

C Beekman, AA Janson, A Baghat, JC van Deutekom… - PloS one, 2018 - journals.plos.org
Duchenne muscular dystrophy (DMD) is a neuromuscular disease characterized by
progressive weakness of the skeletal and cardiac muscles. This X-linked disorder is caused …

Genetics and emerging treatments for Duchenne and Becker muscular dystrophy

N Wein, L Alfano, KM Flanigan - Pediatric Clinics, 2015 - pediatric.theclinics.com
Duchenne and Becker muscular dystrophy (DMD and BMD) are related disorders that occur
because of mutations in the DMD gene, encoding the dystrophin protein. DMD is more …

[HTML][HTML] The D2.mdx mouse as a preclinical model of the skeletal muscle pathology associated with Duchenne muscular dystrophy

DW Hammers, CC Hart, MK Matheny, LA Wright… - Scientific reports, 2020 - nature.com
Duchenne muscular dystrophy (DMD) is an X-linked, lethal muscle degenerative disease
caused by loss of dystrophin protein. DMD has no cure and few treatment options …

[HTML][HTML] A novel high-throughput immunofluorescence analysis method for quantifying dystrophin intensity in entire transverse sections of Duchenne muscular …

V Sardone, M Ellis, S Torelli, L Feng, D Chambers… - PLoS …, 2018 - journals.plos.org
Clinical trials using strategies aimed at inducing dystrophin expression in Duchenne
muscular dystrophy (DMD) are underway or at advanced planning stage, including splice …

Automated immunofluorescence analysis for sensitive and precise dystrophin quantification in muscle biopsies

TA Vetter, S Nicolau, AJ Bradley… - Neuropathology and …, 2022 - Wiley Online Library
Aims Dystrophin, the protein product of the DMD gene, plays a critical role in muscle integrity
by stabilising the sarcolemma during contraction and relaxation. The DMD gene is …

Pathophysiology of duchenne muscular dystrophy: current hypotheses

N Deconinck, B Dan - Pediatric neurology, 2007 - Elsevier
Duchenne muscular dystrophy is a devastating inherited neuromuscular disorder that affects
one in 3300 live male births. Although the responsible gene and its product, dystrophin …

Dystrophin: a clinical perspective

HB Wessel - Pediatric Neurology, 1990 - Elsevier
Dystrophin, the protein product of the gene related to Duchenne and Becker muscular
dystrophies, is a large cytoskeletal protein associated with the muscle fiber membrane …

[HTML][HTML] Mutation-independent proteomic signatures of pathological progression in murine models of Duchenne muscular dystrophy

TLE van Westering, HJ Johansson, B Hanson… - Molecular & Cellular …, 2020 - ASBMB
The absence of the dystrophin protein in Duchenne muscular dystrophy (DMD) results in
myofiber fragility and a plethora of downstream secondary pathologies. Although a variety of …