The muscular dystrophies are commonly associated with cardiovascular complications, including cardiomyopathy and cardiac arrhythmias. These complications are caused by intrinsic defects in ...
In boys with DMD, executive skills such as self-control begin to lag around age 8, but show signs of partial catch-up by 14, ...
Gene therapies have extended survival in muscular dystrophy, requiring improved care transitions from pediatric to adult systems. Current systems lack adequate staffing, specialists, and communication ...
Duchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene. Researchers show how a dual CRISPR RNA method restored dystrophin protein ...
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Setback in gene therapy for Duchenne muscular dystrophy as immune system emerges as key barrier
A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular dystrophy (DMD), after the treatment failed to show significant benefit in a ...
COLUMBUS, Ohio (WCMH) – It’s being called a pioneering event that holds great promise for children who have Duchenne muscular dystrophy. After decades of research, animal studies, and human trials, ...
Pathologic changes of the X-chromosome gene for dystrophin give rise to Duchenne muscular dystrophy (DMD), and dystrophin -- which minimizes muscle fiber loss due to sarcolemma contraction damage when ...
Oculopharyngeal muscular dystrophy (OPMD) is a rare inherited disorder that causes weakness in the eye and throat muscles. It may lead to drooping eyelids and swallowing difficulties. Muscular ...
Becker muscular dystrophy (BMD) is a genetic disorder that causes progressive muscle degeneration and weakness. This can cause a person to have reduced mobility such as problems walking. BMD can make ...
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