Therapeutic approaches to preserve the musculature in Duchenne Muscular Dystrophy: The importance of the secondary therapies
G Angelini, G Mura, G Messina - Experimental Cell Research, 2022 - Elsevier
Muscular dystrophies (MDs) are heterogeneous diseases, characterized by primary wasting
of skeletal muscle, which in severe cases, such as Duchenne Muscular Dystrophy (DMD) …
of skeletal muscle, which in severe cases, such as Duchenne Muscular Dystrophy (DMD) …
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 …
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 …
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …
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] 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 …
by the absence of dystrophin protein, which is essential for muscle fiber integrity. Loss of …
Evolving therapeutic options for the treatment of duchenne muscular dystrophy
ES D'Ambrosio, JR Mendell - Neurotherapeutics, 2023 - Elsevier
Duchenne muscular dystrophy (DMD) is the most common childhood form of muscular
dystrophy. It is caused by mutations in the DMD gene, leading to reduced or absent …
dystrophy. It is caused by mutations in the DMD gene, leading to reduced or absent …
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 …
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 …
mainly encoding cytoskeletal proteins, cause different forms of dystrophy that compromise …
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 …
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 …