Management of children: pharmacological and physical

In the absence of any effective drug treatment, physical methods of management are still the mainstay of treatment. Our current practice in Duchenne muscular dystrophy is to provide lightweight knee-ankle-foot orthoses at the time of loss of ambulation. This prolongs independent walking for an average of two years, and has the effect of delaying the onset of scoliosis, particularly if the boy remains ambulant during the pubertal growth spurt. We are currently assessing the value of radical surgery, performed early in the course of the disease, which may stabilize and prolong independent walking. In non-ambulant patients instrumentation of the spine, using mainly the Luque technique, has revolutionised the treatment of progressive scoliosis. Ventilator support produces clinical improvement in late cases with symptomatic hypoventilation. Its place in the management of asymptomatic patients with nocturnal hypoventilation still needs evaluation, as does the role of early prophylactic respiratory support. We have reviewed the clinical drug trials over the past 10 years. There has been an overall improvement in their quality control.