Integrated Treatment Regime for Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is the most common hereditary neuromuscular disease affecting all races and ethnic groups, with a sex linked inheritance pattern of 1 in 3500 male births. The underlying genetic defect results in the loss of a structural protein called dystrophin that normally serves to provide mechanical stability during muscle contraction. As a result of this loss, there is enhanced myofibre damage/ necrosis, and this forms a prelude to chronic inflammation and significant bystander tissue damage. This destruction of muscle tissue progresses faster than the body’s ability to compensate by regeneration [1,2]. These patients are wheel chair bound by late adolescence and also suffer extensive damage to the cardiac muscles. Death usually occurs as a result of respiratory failure, intractable heart failure or pneumonia [1,2].