[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 …

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 …

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 …

[HTML][HTML] Therapeutic opportunities and clinical outcome measures in Duchenne muscular dystrophy

G Ricci, L Bello, F Torri, E Schirinzi, E Pegoraro… - Neurological …, 2022 - Springer
Introduction Duchenne muscular dystrophy (DMD) is a devastatingly severe genetic muscle
disease characterized by childhood-onset muscle weakness, leading to loss of motor …

Drug discovery of therapies for duchenne muscular dystrophy

Y Blat, S Blat - Journal of biomolecular screening, 2015 - journals.sagepub.com
Duchenne muscular dystrophy (DMD) is a genetic, lethal, muscle disorder caused by the
loss of the muscle protein, dystrophin, leading to progressive loss of muscle fibers and …

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 …

[HTML][HTML] 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 …

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 …

[HTML][HTML] Pharmacological advances for treatment in Duchenne muscular dystrophy

S Guiraud, KE Davies - Current opinion in pharmacology, 2017 - Elsevier
Highlights•Numerous pathophysiological features of DMD provide different therapeutic
avenues.•Pharmacolougical therapies target all muscles and are applicable to all DMD …

[HTML][HTML] Therapeutic strategies for Duchenne muscular dystrophy: an update

C Sun, L Shen, Z Zhang, X Xie - Genes, 2020 - mdpi.com
Neuromuscular disorders encompass a heterogeneous group of conditions that impair the
function of muscles, motor neurons, peripheral nerves, and neuromuscular junctions. Being …