AURORA, Colo. (Dec. 31, 2024) – A groundbreaking study has shed light on the complex interactions between dystrophin, a protein critical to muscle stability, and its partner protein ...
The Shanghai-based firm will test the gene therapy candidate in patients between 4 and 9 years old with Duchenne muscular dystrophy.
Muscular Dystrophy (MD) is a genetic condition causing progressive muscle weakness and complications in vital organs. Early ...
Biophysical characterization of the dystrophin C-terminal domain: Dystrophin interacts differentially with dystrobrevin isoforms. Journal of Biological Chemistry , 2024; 300 (12): 108002 DOI: 10. ...
In 1987, the muscle protein associated with this gene was named dystrophin. Duchenne muscular dystrophy occurs when that gene fails to make dystrophin. Becker muscular dystrophy occurs when a ...
If you have DMD, the gene that makes a protein called dystrophin is broken. This protein normally keeps muscles strong and protects them from injury. The condition is more common in boys because ...
Duchenne muscular dystrophy (DMD) is a rare genetic disorder that is characterised by progressive muscle degeneration and ...
Elevidys, developed by Sarepta Therapeutics, delivers a micro-dystrophin gene designed to compensate for the missing dystrophin-producing genes in patients with DMD. In the trial, "AAV gene ...
It is an X-linked recessive disorder that occurs due to changes in the DMD gene, which codes for the protein dystrophin. Dystrophin is a protein that plays a role in strengthening muscle fibers ...