More than 50 international experts came together last week in a workshop organized by the Parent Project Muscular Dystrophy (PPMD), to discuss bone health and osteoporosis in Duchenne muscular dystrophy (DMD).
The two-day workshop on May 12-13 brought together scientists, clinicians, and industry representatives from around the world. Different aspects of bone health in DMD, including surveillance, monitoring, and treatment were discussed. The effects of glucocorticoids on bone development and strength were also assessed.
“I am honored to work with Leanne Ward, MD, and Lynda Bonewald, PhD, as co-chairs of this event,” said Kathi Kinnett, PPMD’s senior vice president of clinical care, in a press release. “They are both amazing women with long and distinguished careers in pediatric bone health, and both with honors and publications too numerous to list.
“We have worked diligently to develop a compelling and robust agenda, and to convene global experts qualified to dive deeply into important topics around Duchenne bone health and osteoporosis. We look forward to productive and compelling discussions addressing issues that will enhance both quality and quantity of life for those living with Duchenne,” Kinnett added.
DMD is a severe muscle-wasting condition that affects about 1 in every 5,000 boys. Boys with DMD usually lose the ability to walk in their teens and need to use a wheelchair. The condition is caused by a genetic mutation resulting in the absence of a vital muscle protein called dystrophin. But DMD is a multi-system disease affecting many parts of the body, not just muscles, so understanding the effect of the lack of dystrophin on other systems such as bones can help better manage the condition.
Glucocorticoids are the only treatment routinely used in DMD. They are similar to natural hormones produced by the body and reduce inflammation, and may may stabilize or even improve muscle strength for a period of time in some boys. Still, there has been very little research on the effect of glucocorticoids on bone health.
The results of the workshop that was sponsored by Ackerman Foundation, Barbara and Brian Nicholoff, and Catabasis Pharmaceuticals, will be published later in the year.
PPMD is a nonprofit patient organization in the U.S. focused on finding a cure for DMD.