Duchenne muscular dystrophy: an updated review of common available therapies

A Salmaninejad, SF Valilou, H Bayat… - International Journal …, 2018 - Taylor & Francis
Background and purpose: Duchenne muscular dystrophy (DMD) is a lethal progressive
pediatric muscle disorder and genetically inherited as an X-linked disease that caused by …

Emerging genetic therapies to treat Duchenne muscular dystrophy

SF Nelson, RH Crosbie, MC Miceli… - Current opinion in …, 2009 - journals.lww.com
These novel therapies that act to correct the primary genetic defect of dystrophin deficiency
are among the first generation of therapies tailored to correct specific mutations in humans …

Gene therapy for Duchenne muscular dystrophy: an update on the latest clinical developments

C Happi Mbakam, JP Tremblay - Expert Review of …, 2023 - Taylor & Francis
Introduction Duchenne muscular dystrophy (DMD) is one of the most severe and devastating
neuromuscular hereditary diseases with a male newborn incidence of 20 000 cases each …

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 …

Progress toward gene therapy for Duchenne muscular dystrophy

JR Chamberlain, JS Chamberlain - Molecular Therapy, 2017 - cell.com
Duchenne muscular dystrophy (DMD) has been a major target for gene therapy
development for nearly 30 years. DMD is among the most common genetic diseases, 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 …

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 strategies for dystrophin replacement in Duchenne muscular dystrophy

C Happi Mbakam, G Lamothe, JP Tremblay - Frontiers in Medicine, 2022 - frontiersin.org
Duchenne muscular dystrophy (DMD) is an X-linked hereditary disease characterized by
progressive muscle wasting due to modifications in the DMD gene (exon deletions …

Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy

V Himič, KE Davies - European Journal of Human Genetics, 2021 - nature.com
Duchenne muscular dystrophy (DMD) is an X-linked progressive muscle-wasting disorder
that is caused by a lack of functional dystrophin, a cytoplasmic protein necessary for the …

[HTML][HTML] Advances in gene therapy for muscular dystrophies

H Abdul-Razak, A Malerba, G Dickson - F1000Research, 2016 - ncbi.nlm.nih.gov
Duchenne muscular dystrophy (DMD) is a recessive lethal inherited muscular dystrophy
caused by mutations in the gene encoding dystrophin, a protein required for muscle fibre …