Recent advances in gene-editing technologies allow for precise manipulation of the genome to achieve therapeutic effects. While challenges remain, genome editing is likely to one day transform the treatment of muscular dystrophies, according to a review, by scientists from Editas Medicine and Duke University, that highlights current advances…
Northbrook, Illinois-based Marathon Pharmaceuticals, a biopharma that focuses on the development of medicines to treat rare diseases with no current treatment options, has announced it is expanding patient access to its investigational medication deflazacort through its ACCESS DMD program. The program will allow participating physicians to obtain and prescribe deflazacort, which is…
Charles Thornton, M.D., the Saunders Family Distinguished Professor in Neuromuscular Research at the University of Rochester Medical Center’s (URMC) Center for Neural Development & Disease in Rochester, New York, has received a Javits Neuroscience Investigator Award from the National Institutes of Health’s National Institute of Neurological Disorders and…
Radiologic imaging of muscle tissue is increasingly being used as a tool to aid diagnosis of neuromuscular diseases. A review from the University of Padova, Italy, outlines the pros and cons of various radiological techniques in this context. The review, “Role of Radiologic Imaging in Genetic and Acquired Neuromuscular Disorders,“ appeared in the…
Researchers in Spain published a new report describing their search for myotonic dystrophy (DM1) biomarkers. Unfortunately, the search for miRNAs that could help identify the disease was not successful. The report, “Six Serum miRNAs Fail to Validate as Myotonic Dystrophy Type 1 Biomarkers,“ appeared in the journal…
Numerous studies show that deflazacort substantially prolongs walking ability in boys with Duchenne muscular dystrophy (DMD), allowing them to lead a longer productive life. The drug also causes less weight gain than other similar medications, a factor scientists believe might be key to maintaining ambulation. Despite its worldwide popularity as…
The second annual Napa in Newport drew more than 420 guests in a sold-out event that raised more than $700,000 to help to find a cure for Duchenne muscular dystrophy. The money will go to CureDuchenne, a nonprofit dedicated to finding a cure for Duchenne. The event was chaired by Darioush and Shahpar…
Scientists have discovered why fetal muscle stem cells are better at regenerating muscle than adult muscle stem cells. These findings open new avenues for the treatment of muscular dystrophies. Researchers from Sanford Burnham Prebys Medical Discovery Institute, based in La Jolla, California, and Orlando, Florida, investigated the properties of different cells in a study…
Tens of thousands of businesses across the U.S. are participating in this year’s 34th annual Muscular Dystrophy Association (MDA) Shamrocks campaign to raise funds for research and support programs to help people with muscular dystrophy, amyotrophic lateral sclerosis (ALS), and other diseases that severely weaken muscle…
The Precision Medicine Initiative was launched in 2015 to spur biomedical research and the development of a more personalized approach to therapy, one based on an individual’s genetic profile and response to treatment. While the initiative largely focuses on genetically based cancers, a University of Wisconsin-Madison review points out that Emery–Dreifuss muscular…
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