Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy diagnosed in childhood, with approximately 20,000 new cases reported each year. It is a progressive muscle disorder that results ...
A specific gene may play a key role in new treatments that prevent muscle in the body from breaking down in serious muscle diseases, muscular dystrophies. This is shown in a new study at Umeå ...
The Food and Drug Administration (FDA) has approved the first gene therapy for the treatment of Duchenne muscular dystrophy (DMD) in children from age 4 through 5 years of age. Pediatric patients who ...
The MDA's 75th anniversary conference highlights advancements in gene therapy, precision medicine, and patient-centered care for muscular dystrophy. Discussions focus on equitable access to ...
Mutations that lead to muscle atrophy can be repaired with the gene editor CRISPR-Cas9. A team led by ECRC researcher Helena Escobar has now introduced the tool into human muscle stem cells for the ...
Researchers at the University of South Florida USF Health Morsani College of Medicine have identified a potential pathway ...
Phase 3 EPIDYS MRI data quantify givinostat’s potential to preserve functional muscle tissue and reduce fat infiltration in ...
Burn Boot Camp totals $3.8 million in donations to MDA’s mission since 2016. Burn Boot Camp and Muscular Dystrophy ...
The muscular dystrophies are commonly associated with cardiovascular complications, including cardiomyopathy and cardiac arrhythmias. These complications are caused by intrinsic defects in ...
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