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Early Monitoring and Treatment for Bone Health in Duchenne Muscular Dystrophy: Meilan Rutter, MD
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At the 2023 MDA conference, a pediatric endocrinologist from Cincinnati Children’s Hospital talked about the unmet needs in treating bone health for patients with Duchenne muscular dystrophy. [WATCH TIME: 5 minutes]
WATCH TIME: 5 minutes
“I think close monitoring right from the outset is really important. I think that care has moved on from not monitoring that closely to now having more of a proactive approach. We are doing not just DEXA scans, looking at bone density trends, but we're also doing spine X rays routinely looking for spine compression fractures. When those are mild and they can be, you might not even know they're there unless you're looking for them. So monitoring is one [important aspect of care for younger patients] and then treatment.”
Treatment doses of glucocorticoids (GC), with either prednisolone or deflazacort, have had a beneficial impact on the quality of life in boys with Duchenne muscular dystrophy (DMD), mainly through reducing inflammation and preserving muscle function. Although considered a successful treatment option, adverse events of GC include a delay in puberty and bone fragility.1 Thus, continued research developments could potentially lead to a therapy that has a lower risk of adverse effects.
At the recent 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, March 19-22, in Dallas, Texas, Meilan Rutter, MD, chaired a talk session on bone health in DMD and presented on the current state of the field by giving an introduction and setting the stage around the evolution of the current guidelines. The rest of the session, which featured 2 other experts in neuromuscular medicine, focused on bone health including the current management approaches and treatment guidelines. The last talk given highlighted the gaps in research, and future directions beyond the current guidelines.2
At the conference, Rutter, a pediatric endocrinologist from Cincinnati Children’s Hospital, sat down with NeurologyLive® in an interview to provide an overview of what is known currently about bone health in relation to patients with DMD. She also talked about the most important aspects of care for pediatric patients living with DMD and what is still unknown in the field. In addition, Rutter spoke about the challenges patients face in terms of care, such as adverse events from treatment and other unmet needs that should be addressed in the future.
Click here for more coverage of MDA 2023.
