BONE MASS AND BONE METABOLISM IN DUCHENNE MUSCULAR DYSTROPHY

One of the most frequent complications in Duchenne Muscular Dystrophy (DMD) is the increased susceptibility to fractures, causing immobilization and consequent worsening of muscle hypotrophy and weakness. Very few data are available on bone mass and metabolism in DMD patients .although they are frequently given steroids at high dosage to slow the progression of the disease. It is extremely important to discover if the disease per se i.e. before the use of steroids - may induce osteoporosis and bone metabolism derangements, in order to develop an adequate strategy to maximize bone mass and to minimize the skeletal side effects of steroids. Moreover, steroids given early, that is before 6 years of age, when musclefibrosis and degeneration are less severe, could be more effective. Till now the therapy is began at the age of 6 years or later, also because there are no information about the effects of steroids on bone mass and bone metabolism in DMD patients. The aim of this study is to evaluate the evolution bone mass in a group of very young DMD patients (aged 3-6 years), before starting steroid treatment, in order to identify the determinants of bone mass. The study will be carried out by the Istituto Auxologico Italiano IRCCS in collaboration with the Istituto Neurologico Carlo Besta and will involve about 30 patients. We think that a better knowledge of bone mass may reduce the risk of fragility fractures, thus enhancing the quality of life in younger patients affected by Duchenne Muscular Dystrophy, while waiting for a better therapy of this disease.