The muscular dystrophies are commonly associated with cardiovascular complications, including cardiomyopathy and cardiac arrhythmias. These complications are caused by intrinsic defects in ...

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 ...

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 ...

From left: Myosana CEO Matthew Lumley, with co-founders Stanley Froehner and Nick Whitehead. (Myosana and UW Photos) The news: Myosana Therapeutics, a Seattle startup developing gene therapy ...

India, April 5 -- Myotonic dystrophy type 1 (DM1) is the leading form of muscular dystrophy that appears in adulthood. While it is best known for causing progressive muscle weakness and loss, the ...

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 ...

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 ...

Myotonic dystrophy type 1 (DM1) is the most common cause of adult-onset muscular dystrophy, a genetic disorder that leads to muscle weakness and wasting, but also affects the brain, the ...

Researchers from Kyoto University demonstrate how a dual CRISPR RNA method restored dystrophin protein function in induced pluripotent stem cells derived from Duchenne muscular dystrophy (DMD) ...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Epic Bio, a biotechnology company developing therapies to modulate gene expression using compact, non-cutting dCas proteins, today announced a ...