Endocrine and Bone Health Issues | Spinal Muscular Atrophy 2023

This video covers two presentations on endocrine and bone health issues in Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA) patients, especially related to chronic steroid use. Key topics include pathophysiology underlying bone fragility and fractures, current clinical monitoring and treatment approaches focused on bisphosphonates for secondary osteoporosis, impacts on growth and puberty, evolving care considerations, and future directions like preventative fracture strategies and potential benefits from new dissociative steroids like vamorolone. Multiple studies are analyzed quantifying fracture rates, comparing interventions, and assessing drug impacts on bone density and strength. Action items center on earlier monitoring and preventative treatment guided by signs of steroid exposure, more aggressive IV bisphosphonates, assessing vamorolone’s long-term bone effects, and research into primary fracture prevention.