Bone health in adult patients with Duchenne Muscular Dystrophy (DMD). A survey on osteoporosis, fragility fractures and bone-targeted therapies

Duchenne Muscular Dystrophy (DMD) is an X-linked muscle genetic disease leading to loss of independent ambulation in the early teens, restrictive ventilatory insufficiency, and dilatative cardiomyopathy, causing premature death. In the last decade, improvement in standard of care and in the management of cardio-respiratory complications, significantly extended DMD life expectancy and patients can now live up to forty years. In this scenario, bone complications (osteoporosis and fragility fractures) and their management are impacting the quality of life and even mortality with social and economic implications and therefore should be better addressed. As such, with the present proposal - involving several Italian Centers belonging to the DMD Network - we aim to define the bone health status of DMD patients in their adult age. We believe that the generation of robust data on osteoporosis and fragility fractures in these patients will help clinicians to design primary prevention programs and targeted bone-protective clinical trials, and from the patients’ perspectives will improve their quality of life. ultimately reducing the osteoporosis-related social and economic costs.