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 …

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 …

[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 …

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 …

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 …

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] 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 …

[HTML][HTML] Therapy of genetic disorders: novel therapies for Duchenne muscular dystrophy

JT Seto, NE Bengtsson, JS Chamberlain - Current pediatrics reports, 2014 - Springer
Duchenne muscular dystrophy is an inherited, progressive muscle-wasting disorder caused
by mutations in the dystrophin gene. An increasing variety of approaches are moving …

[HTML][HTML] 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 …

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 …