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 …
characterized by progressive deterioration of skeletal muscle that causes rapid loss of …
To skip or not to skip: that is the question for duchenne muscular dystrophy.
MJ Wood - Molecular Therapy: the Journal of the American Society …, 2013 - europepmc.org
GSK/Prosensa's failure doesn't represent undiluted good news given that the utility of exon
skipping is in question and the challenge of designing a study to demonstrate clinical …
skipping is in question and the challenge of designing a study to demonstrate clinical …
[HTML][HTML] 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 …
that is caused by a lack of functional dystrophin, a cytoplasmic protein necessary for the …
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 …
and results from mutations in the dystrophin gene. Currently no treatment is available for this …
[HTML][HTML] Lessons learned from discontinued clinical developments in Duchenne muscular dystrophy
T Markati, L De Waele, U Schara-Schmidt… - Frontiers in …, 2021 - frontiersin.org
Duchenne muscular dystrophy (DMD) is an X-linked condition caused by a deficiency of
functional dystrophin protein. Patients experience progressive muscle weakness …
functional dystrophin protein. Patients experience progressive muscle weakness …
[HTML][HTML] Therapeutic strategies for Duchenne muscular dystrophy: an update
Neuromuscular disorders encompass a heterogeneous group of conditions that impair the
function of muscles, motor neurons, peripheral nerves, and neuromuscular junctions. Being …
function of muscles, motor neurons, peripheral nerves, and neuromuscular junctions. Being …
[图书][B] Duchenne muscular dystrophy: advances in therapeutics
JS Chamberlain, TA Rando - 2006 - books.google.com
Duchenne Muscular Dystrophy (DMD) is one of the most prevalent genetic disorders of
childhood for which there is no cure. This authoritative guide provides a clear overview of …
childhood for which there is no cure. This authoritative guide provides a clear overview of …
Drug discovery of therapies for duchenne muscular dystrophy
Y Blat, S Blat - Journal of biomolecular screening, 2015 - journals.sagepub.com
Duchenne muscular dystrophy (DMD) is a genetic, lethal, muscle disorder caused by the
loss of the muscle protein, dystrophin, leading to progressive loss of muscle fibers and …
loss of the muscle protein, dystrophin, leading to progressive loss of muscle fibers and …
[HTML][HTML] A brief review of Duchenne muscular dystrophy treatment options, with an emphasis on two novel strategies
A Heydemann, M Siemionow - Biomedicines, 2023 - mdpi.com
Despite the full cloning of the Dystrophin cDNA 35 years ago, no effective treatment exists
for the Duchenne Muscular Dystrophy (DMD) patients who have a mutation in this gene …
for the Duchenne Muscular Dystrophy (DMD) patients who have a mutation in this gene …
Progress towards gene therapy for Duchenne muscular dystrophy
MA Hauser, JS Chamberlain - Journal of endocrinology, 1996 - joe.bioscientifica.com
Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder caused by defects in
the dystrophin gene. Patients exhibit progressive muscle degeneration, generally begin …
the dystrophin gene. Patients exhibit progressive muscle degeneration, generally begin …