Pharmacological advances for treatment in Duchenne muscular dystrophy

- Numerous pathophysiological features of DMD provide different therapeutic avenues.
- Pharmacolougical therapies target all muscles and are applicable to all DMD boys.
- Utrophin modulation to compensate for the dystrophin deficiency is in Phase 2 trials.
- Small drugs targeting downstream defects are now in extensive clinical trials.

Duchenne muscular dystrophy (DMD) is a lethal, X-linked muscle-wasting disease caused by lack of dystrophin, essential for muscle fibre integrity. Despite extensive pre-clinical studies, development of an effective treatment has proved challenging. More recently, significant progress has been made with the first drug approval using a genetic approach and the application of pharmacological agents which slow the progression of the disease. Drug development for DMD has mainly used two strategies: (1) the restoration of dystrophin expression or the expression of the compensatory utrophin protein as an efficient surrogate, and (2) the mitigation of secondary downstream pathological mechanisms. This review details current most promising pharmacological approaches and clinical trials aiming to tackle the pathogenesis of this multifaceted disorder.