Immune-mediated pathology in Duchenne muscular dystrophy
AS Rosenberg, M Puig, K Nagaraju… - Science translational …, 2015 - science.org
Immunological and inflammatory processes downstream of dystrophin deficiency as well as
metabolic abnormalities, defective autophagy, and loss of regenerative capacity all …
metabolic abnormalities, defective autophagy, and loss of regenerative capacity all …
Myofibroblasts: trust your heart and let fate decide
J Davis, JD Molkentin - Journal of molecular and cellular cardiology, 2014 - Elsevier
Cardiac fibrosis is a substantial problem in managing multiple forms of heart disease.
Fibrosis results from an unrestrained tissue repair process orchestrated predominantly by …
Fibrosis results from an unrestrained tissue repair process orchestrated predominantly by …
Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy
DE Frank, FJ Schnell, C Akana, SH El-Husayni… - Neurology, 2020 - AAN Enterprises
Objective To report safety, pharmacokinetics, exon 53 skipping, and dystrophin expression
in golodirsen-treated patients with Duchenne muscular dystrophy (DMD) amenable to exon …
in golodirsen-treated patients with Duchenne muscular dystrophy (DMD) amenable to exon …
Duchenne and Becker muscular dystrophies
KM Flanigan - Neurologic clinics, 2014 - neurologic.theclinics.com
The X-linked Duchenne and Becker muscular dystrophies (DMD and BMD) are allelic
disorders occurring due to mutations in the DMD gene, which consists of 79 exons encoding …
disorders occurring due to mutations in the DMD gene, which consists of 79 exons encoding …
[PDF][PDF] AMPK activation regulates LTBP4-dependent TGF-β1 secretion by pro-inflammatory macrophages and controls fibrosis in Duchenne muscular dystrophy
G Juban, M Saclier, H Yacoub-Youssef, A Kernou… - Cell reports, 2018 - cell.com
Chronic inflammation and fibrosis characterize Duchenne muscular dystrophy (DMD). We
show that pro-inflammatory macrophages are associated with fibrosis in mouse and human …
show that pro-inflammatory macrophages are associated with fibrosis in mouse and human …
[HTML][HTML] TGF-β–driven muscle degeneration and failed regeneration underlie disease onset in a DMD mouse model
Duchenne muscular dystrophy (DMD) is a chronic muscle disease characterized by poor
myogenesis and replacement of muscle by extracellular matrix. Despite the shared genetic …
myogenesis and replacement of muscle by extracellular matrix. Despite the shared genetic …
Effect of genetic background on the dystrophic phenotype in mdx mice
WD Coley, L Bogdanik, MC Vila, Q Yu… - Human molecular …, 2016 - academic.oup.com
Genetic background significantly affects phenotype in multiple mouse models of human
diseases, including muscular dystrophy. This phenotypic variability is partly attributed to …
diseases, including muscular dystrophy. This phenotypic variability is partly attributed to …
[HTML][HTML] Natural disease history of the D2-mdx mouse model for Duchenne muscular dystrophy
M van Putten, K Putker, M Overzier… - The FASEB …, 2019 - ncbi.nlm.nih.gov
Abstract The C57BL/10ScSn-Dmd mdx/J (BL10-mdx) mouse has been the most commonly
used model for Duchenne muscular dystrophy (DMD) for decades. Their muscle dysfunction …
used model for Duchenne muscular dystrophy (DMD) for decades. Their muscle dysfunction …
Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study
L Bello, H Gordish-Dressman, LP Morgenroth… - Neurology, 2015 - AAN Enterprises
Objective: We aimed to perform an observational study of age at loss of independent
ambulation (LoA) and side-effect profiles associated with different glucocorticoid …
ambulation (LoA) and side-effect profiles associated with different glucocorticoid …
[HTML][HTML] Fibrillin microfibrils and elastic fibre proteins: Functional interactions and extracellular regulation of growth factors
Fibrillin microfibrils are extensible polymers that endow connective tissues with long-range
elasticity and have widespread distributions in both elastic and non-elastic tissues. They act …
elasticity and have widespread distributions in both elastic and non-elastic tissues. They act …