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

A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular ...

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.

DMD is a progressive, inherited muscle-wasting disease primarily affecting boys, caused by a lack of the protein dystrophin. According to the company, DYNE-251 demonstrated unprecedented ...

Credit: Shutterstock / Dan76. Avidity Biosciences’ Phase I/II trial of ribonucleic acid (RNA) therapy, del-zota, has increased dystrophin in patients with Duchenne muscular dystrophy (DMD). Results ...

DMD is a progressive, inherited muscle-wasting disease primarily affecting boys, caused by a lack of the protein dystrophin.

The determination comes after recruitment and dosing in certain trials were paused after a young patient died due to acute ...

DALLAS—Patients with Duchenne muscular dystrophy with mutations amenable to exon 44 skipping (DMD44) who were treated with delpacibart zotadirsen (AOC 1044) demonstrated statistically significant ...

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 ...

Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory ...