HACKENSACK, N.J., May 12, 2016 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) will convene a Bone Health and Osteoporosis Workshop spanning translational research and clinical care, May 12-13. PPMD has pulled together more than 50 international experts in both pediatric and Duchenne bone health surveillance/monitoring/treatment to discuss a wide range of topics.
Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 5,000 live male births. PPMD has a long history of exploring Duchenne, both from an organ-specific perspective, as well as systemically, throughout the lifespan. Bone health has long been a confusing and often neglected aspect of Duchenne care. There is very little knowledge of underlying bone health in Duchenne, which is further complicated by the possible effects of glucocorticoids on both bone development and bone density/strength. This lack of research and information has resulted in many questions that will be explored over the two days.
Some of the questions and topics that will be explored include:
- What is the underlying effect of Duchenne on bone?
- Duchenne itself may cause changes in bone health and density/strength, which worsens with loss of ambulation. There have been a few studies that have demonstrated bone weakness/fractures in steroid naïve patients that worsen with age.
- What impact does glucocorticoid use have on bone development and density/strength?
- There have been a few studies looking at glucocorticoid use in Duchenne and increased fracture rates. Increased fracture rates are a sign of weakened bones. Unfortunately, long bone fractures often result in the loss of ambulation, so knowledge of the impact of glucocorticoids (types of glucocorticoids, doses, regimes, etc.) on bone is a big issue.
- We have no knowledge about how novel Duchenne therapies, including proposed steroid replacement drugs, might affect bone health. This is an area of needed research.
- How should bone health/density/strength be monitored?
- DEXA scans have been the mainstay of bone health monitoring, but all endocrinologists/centers are not using this technology in the same way. Many providers do not use the DEXA numbers to treat (instead they use fractures and pain); some treat solely using DEXA numbers. No studies have shown that either way is best.
- When should bone health medications (bisphosphonates) be started?
- Some centers start bisphosphonates when they start steroids; some watch the DEXA numbers; some wait for fractures and/or pain. There is no agreed upon protocol and no studies to demonstrate that one plan is better than the other.
- Are there other agents, other than bisphosphonates, that might be better for bone health in Duchenne?
Kathi Kinnett, MSN, CNP, PPMD's Senior Vice President of Clinical Care is excited to bring together this incredible group of experts and believes this meeting will help pull together a set of recommendations and guidelines that will ensure people with Duchenne are receiving optimal, harmonized bone health care. "I am honored to work with Leanne Ward, MD, and Lynda Bonewald, PhD as co-chairs of this event. 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."
The meeting is being sponsored in part by: Ackerman Foundation, Barbara and Brian Nicholoff, and Catabasis Pharmaceuticals.
About Parent Project Muscular Dystrophy
Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne.
We invest deeply in treatments for this generation of young men affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite and educate the global Duchenne community.
Everything we do—and everything we have done since our founding in 1994—helps boys with Duchenne live longer, stronger lives. We will not rest until every young man has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne.
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SOURCE Parent Project Muscular Dystrophy