The OPTIMIZE DMD Consortium is a collaborative network of expert endocrinologists and neurologists, partnered with Parent Project Muscular Dystrophy (PPMD), working to establish a learning health system. This initiative brings together leading clinicians and scientists with the shared mission to advance standards of care, research, education, and advocacy—ultimately aiming to improve contemporary endocrine and bone health outcomes in individuals with Duchenne muscular dystrophy (DMD).

The vision for the OPTIMIZE DMD Consortium is to create a learning health network of expert clinicians and scientists who will work to advance standard of care, research, education and advocacy in order to move the dial on contemporary “endocrine and bone” clinical outcomes in Duchenne muscular dystrophy (DMD). This effort is fueled by the fact that despite significant advances in muscle-targeted therapy for DMD, long-term, high-dose glucocorticoids (GC) remain the backbone of treatment for the foreseeable future. While beneficial to muscle, there are serious consequences of GC therapy in the DMD setting. These include unexpected mortality arising from fat embolism syndrome after a fracture or sub-clinical bone injury, unexpected death due to unrecognized and therefore untreated GC-induced adrenal suppression, premature loss of ambulation following osteoporotic fractures and/or excess weight gain, adverse cardiometabolic effects resulting from GC-driven weight gain, the psychosocial impact of excess weight and profound growth and pubertal delay, and the burden to individuals and families arising from the need to monitor and treat the endocrine complications of GC therapy.