Duchenne muscular dystrophy

D Duan, N Goemans, S Takeda, E Mercuri… - Nature Reviews …, 2021 - nature.com
Duchenne muscular dystrophy is a severe, progressive, muscle-wasting disease that leads
to difficulties with movement and, eventually, to the need for assisted ventilation and …

Duchenne muscular dystrophy: Disease mechanism and therapeutic strategies

A Bez Batti Angulski, N Hosny, H Cohen… - Frontiers in …, 2023 - frontiersin.org
Duchenne muscular dystrophy (DMD) is a severe, progressive, and ultimately fatal disease
of skeletal muscle wasting, respiratory insufficiency, and cardiomyopathy. The identification …

Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo …

CM McDonald, E Marbán, S Hendrix, N Hogan… - The Lancet, 2022 - thelancet.com
Summary Background Cardiosphere-derived cells (CDCs) ameliorate skeletal and cardiac
muscle deterioration in experimental models of Duchenne muscular dystrophy. The HOPE-2 …

Drug development progress in duchenne muscular dystrophy

J Deng, J Zhang, K Shi, Z Liu - Frontiers in Pharmacology, 2022 - frontiersin.org
Duchenne muscular dystrophy (DMD) is a severe, progressive, and incurable X-linked
disorder caused by mutations in the dystrophin gene. Patients with DMD have an absence of …

Update in Duchenne and Becker muscular dystrophy

MA Waldrop, KM Flanigan - Current opinion in neurology, 2019 - journals.lww.com
The mainstay of treatment remains meeting well defined standards of care that have been
shown to influence morbidity and mortality. These include use of systemic steroids, early …

Duchenne muscular dystrophy: Current treatment and emerging exon skipping and gene therapy approach

G Patterson, H Conner, M Groneman, C Blavo… - European Journal of …, 2023 - Elsevier
Duchenne muscular dystrophy (DMD) is an X-linked recessive neuromuscular disorder that
causes debilitating muscle weakness and atrophy due to a loss of the dystrophin protein …

Pharmacology and toxicology of eteplirsen and SRP-5051 for DMD exon 51 skipping: an update

O Sheikh, T Yokota - Archives of Toxicology, 2022 - Springer
Duchenne muscular dystrophy (DMD) afflicts 1 in 5000 newborn males, leading to
progressive muscle weakening and the loss of ambulation between the ages of 8 and 12 …

[HTML][HTML] Cardiomyopathy in Duchenne Muscular Dystrophy and the Potential for Mitochondrial Therapeutics to Improve Treatment Response

S Gandhi, HL Sweeney, CC Hart, R Han, CGR Perry - Cells, 2024 - mdpi.com
Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disease caused by
mutations to the dystrophin gene, resulting in deficiency of dystrophin protein, loss of …

Treatment strategies for cardiomyopathy in children: a scientific statement from the American Heart Association

C Bogle, SD Colan, SD Miyamoto, S Choudhry… - Circulation, 2023 - Am Heart Assoc
This scientific statement from the American Heart Association focuses on treatment
strategies and modalities for cardiomyopathy (heart muscle disease) in children and serves …

One in five patients with Duchenne muscular dystrophy dies from other causes than cardiac or respiratory failure

L Wahlgren, AK Kroksmark, M Tulinius… - European Journal of …, 2022 - Springer
Duchenne muscular dystrophy (DMD) is a severe neuromuscular disorder with increasing
life expectancy from late teens to over 30 years of age. The aim of this nationwide study was …