Emerging genetic therapies to treat Duchenne muscular dystrophy
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 …
are among the first generation of therapies tailored to correct specific mutations in humans …
Duchenne muscular dystrophy: an updated review of common available therapies
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 …
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 …
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 …
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 …
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 …
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 …
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 …
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 …
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 …
preclinical and clinical trials, there remains considerable interest in the development of …