Recent advances in the treatment of Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is an x-linked, progressive, incurable disease which affects approximately 1 in 3,500 –5,000 live boy births. The condition is caused by a lack of a functional protein, dystrophin, in the muscle. Exciting new advances have been made in the treatment of this condition, including genetic treatments. Treatments for DMD have improved over the last 5–10 years, both a combination of im proved standards of care and equity across treating centres as well as access to clinical trials and some treatments that have been approved as a result of clinical trials.
Source: Paediatrics and Child Health - Category: Pediatrics Authors: Tags: Symposium: Neurodisability Source Type: research