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New Drug Shows Promise for Treating Duchenne Muscular Dystrophy

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Drug,Vamorolone,Duchenne Muscular Dystrophy

Researchers have developed a new drug called vamorolone that shows promise in treating Duchenne Muscular Dystrophy (DMD), the most common genetic disease. The drug aims to address the loss of the dystrophin protein in muscle tissues, which leads to progressive weakness and challenges with day-to-day activities. Vamorolone is designed to dissociate the benefits of glucocorticoid drugs from their side effects, such as bone and growth disturbances.

DMD is the most common genetic disease. It leads to the loss of the dystrophin protein in muscle tissues, with progressive weakness and challenges with day-to-day activities. The DMD gene is the largest gene in the human genome, spanning 2,300,000 base pairs on the X chromosome. Because the gene is on the X chromosome, mostly young boys are affected.

Kanneboyina Nagaraju, professor of pharmaceutical sciences and dean of the School of Pharmacy and Pharmaceutical Sciences, Binghamton University Related StoriesHoffman, who has been working on translational research on DMD since 1985, established the Cooperative International Neuromuscular Research Group to enable robust clinical trials in DMD. Vamorolone became one of the first drugs tested by the CINRG international clinical trial network. In a double-blind placebo-controlled trial in 121 DMD boys, ages 4 to 7, vamorolone was found to increase strength and mobility relative to placebo.

 

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