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…
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…
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…
Pfizer, in collaboration with Newcastle University, reports finding four serum biomarkers for three types of muscular dystrophy. The markers may be valuable for monitoring disease progression and treatment response in both preclinical and clinical studies. Researchers analyzed previously collected serum samples from three types of muscular dystrophies — 38 Becker muscular dystrophy (BMD) and…
UCLA scientists have developed a potential gene therapy approach for Duchenne muscular dystrophy (DMD) using CRISPR/Cas9 technology and stem cells. If the new treatment proves successful and reaches the clinic — possibly in the next decade — it could be applied to 60 percent of all Duchenne patients. CRISPR/Cas9 is a gene-editing technology that allows targeting and…
A discovery clears up the confusion about the molecular factors involved in muscle repair. The research, published in the journal e-life, shows that a protein complex containing the factors TBP and TFIID is essential to activate genes responsible for adult muscle tissue regeneration. Adult muscles contain stem cells that can be activated…
Catabasis Pharmaceuticals announced it will present data on CAT-1004 at the XIV International Conference on Duchenne and Becker Muscular Dystrophy to be held in Rome, Italy, on Feb. 13-14. Joanne Donovan, chief medical officer of Catabasis, will talk about the drug, now in clinical testing, and the design of the MoveDMD…
Sarepta Therapeutics, a developer of RNA-targeted treatments, announced that the U.S. Food and Drug Administration (FDA) is extending by another three months its review of the company’s New Drug Application (NDA) for eteplirsen, a potential treatment for Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. A reason for the extension was…
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