Advances in stem cell therapy using patients’ own cells with genetically modified dystrophin have been hampered by the large size of the dystrophin gene, which are difficult to pack into a delivery system. Now, scientists at University College London have managed to produce a gene construct much bigger than was…
Regenerative medicine is a branch of research that deals with the process of replacing, engineering or regenerating human cells, tissues, or organs to restore or establish normal function. But a major challenge in this field has been delivering treatments to control cellular behavior. One possible strategy to overcome this problem is to…
Comedian and actor Jerry Lewis, the longtime Muscular Dystrophy Association national chairman and host of the organization’s former Labor Day Telethon, is returning from his retired role with the groundbreaking charity that revolutionized public philanthropic giving in America. Lewis helped introduce MDA’s revitalized brand on Jan. 29, when the Chicago-based charity…
Akashi Therapeutics announced they are suspending both dosing and patient enrollment in the HALO trial – a study investigating their experimental therapy, HT-100, in patients with Duchenne muscular dystrophy. The trial’s suspension came after a patient enrolled in the highest dose-group of the study began experiencing life-threatening adverse effects. At this time,…
Catabasis Pharmaceuticals, Inc., recently announced that the first part of its MoveDMD clinical trial, a Phase 1/2 study of CAT-1004 in the treatment of Duchenne muscular dystrophy (DMD), has received positive top-line results, and it will soon begin the study’s second phase. “We are pleased with these results and believe that…
Duke University graduate student Mark Juhas is engineering a highly functional skeletal muscle tissue that has shown great promise and might constitute a valuable model for treatment of muscular dystrophy and other disorders, such as myositis, the inflammation and deterioration of the muscle tissue. According to the National Institute of Neurological…
Duke University researcher Brent Hoffman is investigating the mechanical nature of major diseases — a research area named mechanobiology — including muscular dystrophy. Hoffman’s research could lead to the development of new treatment approaches for mechanosensitive diseases. Many complicated diseases have a little understood mechanical component. Dr. Hoffman, an assistant…
Sarepta Therapeutics, Inc., announced that a U.S. Food and Drug Administration (FDA) advisory committee has been forced to postpone Friday’s scheduled hearing to review the company’s New Drug Application (NDA) for eteplirsen, a potential treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. A severe winter storm starting…
Researchers have found that RNA sequencing is able to identify disease-causing genetic mutations in non-coding regions of the DNA. The technique can be used to aid in diagnosing muscular dystrophy (MD). The study, “RNAseq analysis for the diagnosis of muscular dystrophy,“ appeared in the journal Annals of Clinical and Translational…
Lochte Insurance Agency is organizing a charity drive to raise awareness and support for the San Antonio Muscle Walk on April 9, an annual event sponsored by the Muscular Dystrophy Association (MDA). The company’s goal is to pay for one child to attend the MDA Camp. The Lochte agency is…
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