Saturday, May 8, 2010

Treatment Options for Duchenne Muscular Dystrophy

There is neither a medical cure for this disease nor a method of slowing its progression. Much can be done to treat complications and to improve the quality of life of affected children.


Cardiac decompensation often responds initially well to digoxin.
Pulmonary infections should be promptly treated. Patients should avoid contact with children who have obvious respiratory or other contagious illnesses. Immunizations for influenza virus and other routine vaccinations are indicated.
Preservation of a good nutritional state is importantBecause sedentary children burn fewer calories than active children and because of depression as an additional factor, these children tend to eat excessively and gain weight. Obesity makes a patient with myopathy even less functional because part of the limited reserve muscle strength is dissipated in lifting the weight of excess subcutaneous adipose tissue. Dietary restrictions with supervision may be needed.
Physiotherapy delays but does not always prevent contractures.

Other treatment of human patients with Duchenne dystrophy involves the use of prednisone, prednisolone, deflazacort, or other steroids. Glucocorticoids decrease the rate of apoptosis or programmed cell death of myotubes during ontogenesis and may decelerate the myofiber necrosis in muscular dystrophy
One protocol gives prednisone (0.75 mg/kg/day) for the first 10 days of each month to avoid
chronic complications

Nevertheless, some cases of Duchenne dystrophy treated early with steroids appear to have an improved long-term prognosis as well as the short-term improvement and may help keep patients ambulatory for more years than expected if untreated

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