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Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene expressed in differentiated myofibers. Researchers at The Ottawa Hospital and the University of Ottawa recently discovered that muscle stem cells also express dystrophin, affecting the generation of functional muscle fibers. Their discovery could revolutionize understanding of the disease and eventually…

BioBlast Pharma Ltd. has announced positive results from its Phase 2 open label clinical trial, HOPEMD, of Cabaletta (IV trehalose) in 25 patients suffering from oculopharyngeal muscular dystrophy (OPMD). OPMD is an uncommon disease in which progressive muscle-wasting occurs. People with OPMD have severe problems with swallowing (dysphagia), which can cause them…

Lipid-lowering medications are used by many people to reduce risk of heart disease and other medical conditions. A new study from Seattle, Washington, indicates an unexpected use of simvastatin, commonly known by the brand-name Zocor in the United States. According to the report, simvastatin may help skeletal muscles become more healthy in…

Cellular models can elucidate molecular mechanisms underlying several diseases, reducing animal usage in biomedical sciences and following the guiding principles for animal testing. Now, researchers at the Karolinska Institute and at the Karolinska University published a study entitled “Primary Murine Myotubes as a Model for Investigating Muscular…

Researchers at the University of California published a study entitled “Human Satellite Cell Transplantation and Regeneration from Diverse Skeletal Muscles” in the journal Stem Cell Reports whereby they reveal isolated human muscle stem cells capable of repairing damaged muscles. One of the major problems of treating…

A new study entitled “GRAF1 deficiency blunts sarcolemmal injury repair and exacerbates cardiac and skeletal muscle pathology in dystrophin-deficient mice” and published in the Skeletal Muscle Journal by Chapel Hill, North Carolina and Northwestern University in Chicago researchers, demonstrates that the GRAF1 protein aids in the repair…

A new report titled “Improving clinical trial design for Duchenne muscular dystrophy“, published in the journal BMC Neurology, suggests that some novel treatments for Duchenne muscular dystrophy (DMD) may have positive effects in those affected by the disease but these results are not showing up in clinical trials due to the way studies are being designed.

New research out of Germany indicates that immune therapy might help treat Duchenne muscular dystrophy (DMD), a serious disease that causes muscle weakness. In a mouse model of the disease, the ability to run, as well as disease biomarkers, appeared to improve following antibody treatment, specifically a form of human…

Britain is now the first country in the world that allows the fusion of DNA from three people for the creation of babies. This could provide a solution for preventing diseases such as muscular dystropy, a hereditary disease characterized by progressive weakening of the muscles. The decision involved a change to in vitro fertilization…

PTC Therapeutics, Inc. submitted a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for Translarna™. The medication is used for treating nonsense mutation Duchenne muscular dystrophy (nmDMD). Duchenne muscular dystrophy (DMD) is a progressive disorder of the muscles that mostly affects males. It is caused by a…