Duchenne muscular dystrophy: pathogenesis and promising therapies

M Chang, Y Cai, Z Gao, X Chen, B Liu, C Zhang… - Journal of …, 2023 - Springer
Duchenne muscular dystrophy (DMD) is a severe, progressive, muscle-wasting disease,
characterized by progressive deterioration of skeletal muscle that causes rapid loss of …

[HTML][HTML] 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 …

Evolving therapeutic options for the treatment of duchenne muscular dystrophy

ES D'Ambrosio, JR Mendell - Neurotherapeutics, 2023 - Elsevier
Duchenne muscular dystrophy (DMD) is the most common childhood form of muscular
dystrophy. It is caused by mutations in the DMD gene, leading to reduced or absent …

Impending therapies for Duchenne muscular dystrophy

TA Partridge - Current opinion in neurology, 2011 - journals.lww.com
Although genetic and cell-mediated approaches are currently showing genuine promise in
preclinical and clinical trials, there remains considerable interest in the development of …

Clinical management of Duchenne muscular dystrophy: the state of the art

S Messina, GL Vita - Neurological Sciences, 2018 - Springer
Introduction Duchenne muscular dystrophy (DMD) is a devastating, progressive
neuromuscular disorder for which there is no cure. As the dystrophin gene is located on the …

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 …

Therapeutic developments for Duchenne muscular dystrophy

IEC Verhaart, A Aartsma-Rus - Nature Reviews Neurology, 2019 - nature.com
Duchenne muscular dystrophy (DMD) is caused by the lack of functional dystrophin protein.
Improvements in patient care and disease management have slowed down disease …

Pharmacological therapeutics targeting the secondary defects and downstream pathology of Duchenne muscular dystrophy

JM Spinazzola, LM Kunkel - Expert opinion on orphan drugs, 2016 - Taylor & Francis
Introduction: Since the identification of the dystrophin gene in 1986, a cure for Duchenne
muscular dystrophy (DMD) has yet to be discovered. Presently, there are a number of …

Therapeutic approaches to preserve the musculature in Duchenne Muscular Dystrophy: The importance of the secondary therapies

G Angelini, G Mura, G Messina - Experimental Cell Research, 2022 - Elsevier
Muscular dystrophies (MDs) are heterogeneous diseases, characterized by primary wasting
of skeletal muscle, which in severe cases, such as Duchenne Muscular Dystrophy (DMD) …

[HTML][HTML] 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 …