with rapid progression from congestive heart failure to death or transplantation. Dr. Jeffrey Towbin and colleagues first identified the disease-causing gene as dystrophin. Dystrophin is a ...

Cardiomyopathy is a disease that weakens the heart muscle. In DMD, low levels of dystrophin protein cause the heart to grow bigger and the heart muscle to weaken. These changes make it harder for ...

Without dystrophin, your muscle cells remain unprotected ... Children who show symptoms at a younger age are at a higher risk of heart problems than those who show symptoms later.

A new article argues dystrophin immunogenicity is an under-examined issue in the treatment of Duchenne muscular dystrophy.

None of the eteplirsen-treated patients reached a left ventricular ejection fraction below 50% compared with 22.1% of patients in the control group.

The study reveals that dystrophin's CT domain interacts differently ... which extend beyond skeletal muscles to affect organs ...

Sustained exon skipping, dystrophin restoration, and improved muscle indicators were observed, including reduced inflammation and fibrosis. A 3.8-second improvement in time to rise (TTR ...

Elevidys was granted expanded approval by the U.S. Food and Drug Administration in 2024, with expectations high that the therapy, designed to restore a critical muscle protein called dystrophin ...

Dystrophin is critical to the structural stability of all muscles, including skeletal, diaphragm, and heart muscles. Patients with Duchenne can lose the ability to walk (loss of ambulation ...