Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: past, present, and future

A Łoboda, J Dulak - Pharmacological Reports, 2020 - Springer
Background Duchenne muscular dystrophy (DMD) is a severe X-linked neuromuscular
childhood disorder that causes progressive muscle weakness and degeneration and results …

Cardiovascular disease in Duchenne muscular dystrophy: overview and insight into novel therapeutic targets

TI Schultz, FJ Raucci Jr, FN Salloum - Basic to Translational Science, 2022 - jacc.org
Duchenne muscular dystrophy (DMD) is a devastating disease affecting approximately 1 in
every 3,500 male births worldwide. Multiple mutations in the dystrophin gene have been …

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 …

[HTML][HTML] Current pharmacological strategies for Duchenne muscular dystrophy

S Yao, Z Chen, Y Yu, N Zhang, H Jiang… - Frontiers in Cell and …, 2021 - frontiersin.org
Duchenne muscular dystrophy (DMD) is a lethal, X-linked neuromuscular disorder caused
by the absence of dystrophin protein, which is essential for muscle fiber integrity. Loss of …

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 …

From diagnosis to therapy in Duchenne muscular dystrophy

A Babbs, M Chatzopoulou, B Edwards… - Biochemical Society …, 2020 - portlandpress.com
Genetic approaches for the diagnosis and treatment of inherited muscle diseases have
advanced rapidly in recent years. Many of the advances have occurred in the treatment of …

Therapeutic approaches for Duchenne muscular dystrophy

TC Roberts, MJA Wood, KE Davies - Nature Reviews Drug Discovery, 2023 - nature.com
Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disorder and a
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …

The multifaceted view of heart problem in Duchenne muscular dystrophy

U Florczyk-Soluch, K Polak, J Dulak - Cellular and Molecular Life Sciences, 2021 - Springer
Dystrophin is a large protein serving as local scaffolding repetitively bridging cytoskeleton
and the outside of striated muscle cell. As such dystrophin is a critical brick primarily in …

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 …

Innovative therapeutic approaches for Duchenne muscular dystrophy

F Fortunato, R Rossi, MS Falzarano… - Journal of Clinical …, 2021 - mdpi.com
Duchenne muscular dystrophy (DMD) is the most common childhood muscular dystrophy
affecting~ 1: 5000 live male births. Following the identification of pathogenic variations in the …