Dr. I. Ginjaar, LUMC Leiden
Dr. I. Groot, UMC St Radboud Nijmegen

There is no cure for DMD. Treatment goals are to maintain quality of life, by means of physiotherapy interventions, social and psychological support, aids and devices. Each phase of the disease has specific treatment aims. Four different phases can be discerned: the ambulant phase, the early wheelchair phase, the phase of cardiac and lung complications and the adult phase. In the beginning more emphasize will lay on the physical treatment, while this will gradually change to social support and participation in society. Monitoring nutrition is advised, not only for over or underweight, but also for dietary calcium intake with regard to osteoporosis, especially when corticosteroids are taken. Scoliosis can not be prevented, but the incidence is decreasing since the use of corticosteroids. If a scoliosis is progressive, surgical correction is the only effective way to straighten the spine.

Steroids do have a beneficial effect on muscle force and muscle function, and functional abilities. In the Netherlands a protocol for the use of corticosteroids in boys with DMD is available; the most widely used regimen in the Netherlands is 10 days use of corticosteroids (0.75 mg/kg/d in the ambulant phase, and 0.5 mg/kg/d in the wheelchair phase). A minor population is taking daily corticosteroids (0.75 mg/kg/d). If a boy takes corticosteroids he should be closely monitored for side effects, especially osteoporosis and vertebral deformity.  

Other treatments aiming at the correction of the gene defect itself are not yet available for clinical use, however promising trials with DMD boys are ongoing.

- Treat-NMD Standards of care voor Duchenne spierdystrofie. 2010. Open here
- Richtlijn: Het gebruik van corticosteroiden bij Duchenne spierdystrofie. December 2004. Open here