[HTML][HTML] Emerging strategies in the treatment of Duchenne muscular dystrophy

PB Shieh - Neurotherapeutics, 2018 - Elsevier
Duchenne muscular dystrophy (DMD) is a progressive X-linked degenerative muscle
disease due to mutations in the DMD gene. Genetic confirmation has become standard in …

Molecular treatments in Duchenne muscular dystrophy

M Guglieri, K Bushby - Current opinion in pharmacology, 2010 - Elsevier
Duchenne muscular dystrophy (DMD) is the most common childhood muscular dystrophy
and results from mutations in the dystrophin gene. Currently no treatment is available for this …

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 …

Rehabilitation management of the patient with Duchenne muscular dystrophy

LE Case, SD Apkon, M Eagle, A Gulyas, L Juel… - …, 2018 - publications.aap.org
Steadily improving management of Duchenne muscular dystrophy (DMD) continues to lead
to improved physical and functional status, allowing increasingly successful transitions to …

Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches

RJ Fairclough, MJ Wood, KE Davies - Nature Reviews Genetics, 2013 - nature.com
Duchenne muscular dystrophy (DMD) is a devastating progressive disease for which there
is currently no effective treatment except palliative therapy. There are several promising …

New therapies for Duchenne muscular dystrophy: challenges, prospects and clinical trials

G Cossu, M Sampaolesi - Trends in molecular medicine, 2007 - cell.com
Muscular dystrophies primarily affect skeletal muscle. Mutations in a large number of genes,
mainly encoding cytoskeletal proteins, cause different forms of dystrophy that compromise …

[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 …

Emerging drugs for Duchenne muscular dystrophy

V Malik, LR Rodino-Klapac… - Expert opinion on …, 2012 - Taylor & Francis
Introduction: Duchenne muscular dystrophy (DMD) is the most common, severe childhood
form of muscular dystrophy. Treatment is limited to glucocorticoids that have the benefit of …

The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review

S Ryder, RM Leadley, N Armstrong… - Orphanet journal of rare …, 2017 - Springer
Abstract Background Duchenne Muscular Dystrophy (DMD) is a rapidly progressive, lethal
neuromuscular disorder, present from birth, which occurs almost exclusively in males. We …

[HTML][HTML] Combined therapies for Duchenne muscular dystrophy to optimize treatment efficacy

G Cordova, E Negroni, C Cabello-Verrugio… - Frontiers in …, 2018 - frontiersin.org
Duchene Muscular Dystrophy (DMD) is the most frequent muscular dystrophy and one of the
most severe due to the absence of the dystrophin protein. Typical pathological features …