Consensus concept of modern effective therapy for Duchenne muscular dystrophy

Duchenne muscular dystrophy is a genetic orphan neuromuscular disease caused by mutation in the DMD gene encoding protein dystrophin. As result of developing and progressive muscle damage atrophy, children lose ability to walk, develop respiratory cardiac disorders. The core elements good care standards are early diagnosis, prevention treatment osteoporosis, daily physical therapy, regular rehabilitation, glucocorticosteroids, control heart lung function. clinical effect new targeted pathogenetic therapies for dystrophy, restoring synthesis full or truncated dystrophin, depend on their appropriate combination with existing care.