Duchenne muscular dystrophy: from diagnosis to therapy

MS Falzarano, C Scotton, C Passarelli, A Ferlini - Molecules, 2015 - mdpi.com
Duchenne muscular dystrophy (DMD) is an X-linked inherited neuromuscular disorder due
to mutations in the dystrophin gene. It is characterized by progressive muscle weakness and …

Cardiac pathophysiology and the future of cardiac therapies in Duchenne muscular dystrophy

TA Meyers, DW Townsend - International journal of molecular sciences, 2019 - mdpi.com
Duchenne muscular dystrophy (DMD) is a devastating disease featuring skeletal muscle
wasting, respiratory insufficiency, and cardiomyopathy. Historically, respiratory failure has …

[HTML][HTML] TGF-β–driven muscle degeneration and failed regeneration underlie disease onset in a DMD mouse model

DAG Mázala, JS Novak, MW Hogarth, M Nearing… - JCI insight, 2020 - ncbi.nlm.nih.gov
Duchenne muscular dystrophy (DMD) is a chronic muscle disease characterized by poor
myogenesis and replacement of muscle by extracellular matrix. Despite the shared genetic …

Stem cell-based therapies for Duchenne muscular dystrophy

C Sun, C Serra, G Lee, KR Wagner - Experimental neurology, 2020 - Elsevier
Muscular dystrophies are a group of genetic muscle disorders that cause progressive
muscle weakness and degeneration. Within this group, Duchenne muscular dystrophy …

Current and emerging treatment strategies for Duchenne muscular dystrophy

JK Mah - Neuropsychiatric disease and treatment, 2016 - Taylor & Francis
Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy in
childhood. It is caused by mutations of the DMD gene, leading to progressive muscle …

ER stress in skeletal muscle remodeling and myopathies

D Afroze, A Kumar - The FEBS Journal, 2019 - Wiley Online Library
Skeletal muscle is a highly plastic tissue in the human body that undergoes extensive
adaptation in response to environmental cues, such as physical activity, metabolic …

Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases

MK Tsoumpra, S Fukumoto, T Matsumoto, S Takeda… - …, 2019 - thelancet.com
Duchenne muscular dystrophy (DMD) is an X-linked disorder characterized by progressive
muscle degeneration, caused by the absence of dystrophin. Exon skipping by antisense …

Wnt signaling in skeletal muscle dynamics: myogenesis, neuromuscular synapse and fibrosis

P Cisternas, JP Henriquez, E Brandan… - Molecular …, 2014 - Springer
The signaling pathways activated by Wnt ligands are related to a wide range of critical cell
functions, such as cell division, migration, and synaptogenesis. Here, we summarize …

Complexity of skeletal muscle degeneration: Multi-systems pathophysiology and organ crosstalk in dystrophinopathy

K Ohlendieck, D Swandulla - Pflügers Archiv-European Journal of …, 2021 - Springer
Duchenne muscular dystrophy is a highly progressive muscle wasting disorder due to
primary abnormalities in one of the largest genes in the human genome, the DMD gene …

Current understanding of molecular pathology and treatment of cardiomyopathy in duchenne muscular dystrophy

TLE Van Westering, CA Betts, MJA Wood - Molecules, 2015 - mdpi.com
Duchenne muscular dystrophy (DMD) is a genetic muscle disorder caused by mutations in
the Dmd gene resulting in the loss of the protein dystrophin. Patients do not only experience …