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

Dystrophin is part of a protein complex linking the cytoskeleton of muscle fibers to the surrounding connective tissue (basal lamina). It’s a long protein with numerous redundant coils (purple balls), ...

One gene, for instance, controls the production of dystrophin, a protein that protects muscle fibers from damage. A change in this gene results in limited or discontinued dystrophin production.

Avidity Biosciences’ Phase I/II trial of ribonucleic acid (RNA) therapy, del-zota, has increased dystrophin in patients with Duchenne muscular dystrophy (DMD). Results from the company’s ...

These include gene-based therapies (e.g., replacing a patient's faulty DMD genes with normally functioning ones), cell-based therapies (e.g., replacing dystrophin-deficient muscle cells with stem ...

It affects about one in 3,300 boys. Delandistrogene moxeparvovec delivers a gene that codes for a shortened form of ...