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New Study Insights Hold Potential For Kids Suffering From DMD

The present standard of care employed to treat children with DMD (Duchenne Muscular Dystrophy), Prednisone, lowers chronic inflammation but has severe consequence. Eplerenone, a heart failure medicine, is employed in elderly population to cure cardiomyopathy (a leading cause of death for people suffering from DMD). A new medicine in progress seems to merge the advantageous effects of these drugs for the muscle and heart while also displaying enhanced security in experimental models. This drug, vamorolone, does so by concurrently aiming for 2 nuclear receptors essential in controlling cardiomyopathy and inflammation. This is shown in a small study posted in Life Science Alliance.

DMD is a progressive X-connected disease that takes place mostly in men. It is distinguished by muscle weakness that gets worse eventually, and most children with DMD will employ wheelchairs by the time they are teens. DMD is caused by DMD gene mutation. This gene offers instructions for creating dystrophin, a protein discovered mostly in respiratory, skeletal, and heart muscles.

On a related note, in an evaluation of registry info for over 5,300 liver transplants conducted in kids all over the nation, researchers at Johns Hopkins Medicine verify the kind of patient who is most expected to stay alive in a split liver transplant (getting just a part of a liver) with no extra long-term health dangers, which can permit for an elevation in the accessibility of organs. A report on the new research is posted in the Liver Transplantation journal’s December issue.

“Children dying while on the waitlist are the worst probable outcome,” claims Douglas Mogul to the media in an interview. He is Pediatric Liver Transplant Program’s medical director at Johns Hopkins Children Center. He is also an assistant professor of pediatric hepatology, gastroenterology, and nutrition at the Johns Hopkins University School of Medicine.

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