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

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.

The improvement in dystrophin was approximately in line with the 5.9% unadjusted improvement seen with Viltepso in a phase 2 study that supported its 2020 approval by the FDA. Shares in Wave rose ...

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 ...

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

Treatment with the antibody oligonucleotide conjugate increased dystrophin production by 25%, the San Diego biotech shared in August, which restored total dystrophin levels by 58%. The treatment ...

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 ...

Biopsy data from eight of the boys showed a 6.4% mean muscle content-adjusted dystrophin. The average dystrophin between week 24 and 48 was 7.8%, the company said. Seven of these eight boys ...