Duchenne Muscular Dystrophy: diagnosis, clinical development and global researchMuscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, ...

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.

DMD is an X-linked disorder characterized by progressive muscle weakness and wasting due to the absence of a protein called dystrophin, which in turn causes degeneration of skeletal and cardiac muscle ...

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 ...

The findings suggest that the body's own immune system may be undermining the treatment with gene therapy, such as Elevidys ...

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 ...

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 ...