Hormonal therapy in patients with Duchenne-type muscular dystrophy

        Duchenne muscular dystrophy (DMD) is the most common type of muscular dystrophy with the worst prognosis, and is the most common x-linked recessive genetic myopathy in China, with an incidence of about 3/100,000 live births of male infants, and foreign reports of 1/3600-6000 live births of male infants; it is a lethal disease. The average life expectancy of affected children was only 14.4 years in the 1960s.        Although the molecular genetics of DMD has been clarified for decades, and gene therapy research based on this foundation has proliferated, the problem of etiologic treatment has not been solved, and there is no effective cure for DMD. However, with the use of glucocorticosteroids, the average life expectancy of children was extended to 19.3 years in 1990 and 24.5 years in 2012 due to the use of other comprehensive complementary treatments such as non-invasive ventilation. It is the only pharmacological treatment that has so far proven effective in delaying disease progression.        Although glucocorticosteroids have numerous side effects and some are even difficult to tolerate and have to be reduced or discontinued, they are still a mainstay in the treatment of the disease under current conditions. There is ample evidence that glucocorticosteroids are recommended for all patients with DMD to preserve ambulatory function and reduce cardiac, pulmonary, and skeletal complications for as long as possible.