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

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, ...

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.

A few weeks after Sarepta Therapeutics confirmed a patient died after receiving its drug Elevidys, research published April 3 ...

A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments.

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

Avidity Biosciences’ Phase I/II trial of ribonucleic acid (RNA) therapy, del-zota, has increased dystrophin in patients with ...

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 ...

About Duchenne Muscular Dystrophy DMD is a progressive neuromuscular disorder caused by a mutation in the DMD gene which affects the production of a protein called dystrophin. 3 Dystrophin is a ...