ARMGO Pharma and the Muscular Dystrophy Association today announced that $1 Million has been awarded for preclinical work in support of an Investigational New Drug application with the U.S. Food and Drug Administration for ARM210, a novel, orally available, small-molecule Rycal drug that has potential as a treatment for Duchenne muscular dystrophy.
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Royal Holloway University, is today giving money generated by its research into Duchenne muscular dystrophy to the Muscular Dystrophy Campaign.
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DART Therapeutics Inc., an innovative, new-model biotechnology firm focused on developing therapies for Duchenne muscular dystrophy, announced today that it is developing a SARM drug candidate obtained from Belgium-based Galapagos NV.
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The following research from Columbia University Medical Center and NewYork-Presbyterian Hospital is being presented at the 65th annual meeting of the American Academy of Neurology, March 16-23, 2013, in San Diego.
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A genetic mutation that alters the kinetics of an ion channel in red blood cells has been identified as the cause behind a hereditary anemia, according to a paper published this month in the Proceedings of the National Academy of Sciences by University at Buffalo scientists and colleagues.
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Researchers at the University of Minnesota's Lillehei Heart Institute have combined genetic repair with cellular reprogramming to generate stem cells capable of muscle regeneration in a mouse model for Duchenne Muscular Dystrophy (DMD).
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Steroid therapy is associated with a considerable reduction in all-cause mortality and new-onset and progressive cardiomyopathy in patients with the debilitating X-linked disease Duchenne muscular dystrophy.
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A multi-million Euro initiative is bringing together researchers from across the world to develop new diagnostic tools and new treatments for people with rare diseases and to connect research data in this area on a global scale.
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Muscular dystrophy is caused by the largest human gene, a complex chemical leviathan that has confounded scientists for decades. Research conducted at the University of Missouri and described this month in the Proceedings of the National Academy of Sciences has identified significant sections of the gene that could provide hope to young patients and families.
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Parent Project Muscular Dystrophy (PPMD) has awarded Dr. Craig McDonald of the University of California, Davis (UC Davis) $175,000 in supplemental funds to expand his ongoing study through the 20 CINRG (Cooperative International Neuromuscular Research Group) centers to better understand the progression of Duchenne muscular dystrophy (Duchenne) and determine the impact of the Duchenne standards of care established by the Centers for Disease Control (CDC).
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Researchers have found that injecting aorta-derived stem cells into the hearts of dystrophin-deficient mice prevents the onset of dilated cardiomyopathy, raising hopes of a potential new treatment approach to prevent or reverse the condition in human Duchenne muscular dystrophy.
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Usually, results from a new study help scientists inch their way toward an answer whether they are battling a health problem or are on the verge of a technological breakthrough. Once in a while, those results give them a giant leap forward. In a preliminary study in a canine model of Duchenne muscular dystrophy (DMD), University of Missouri scientists showed exactly such a leap using gene therapy to treat muscular dystrophy.
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A new study has found that tamoxifen, a well-known breast cancer drug, can counteract some pathologic features in a mouse model of Duchenne muscular dystrophy (DMD). At present, no treatment is known to produce long-term improvement of the symptoms in boys with DMD, a debilitating muscular disorder that is characterized by progressive muscle wasting, respiratory and cardiac impairments, paralysis, and premature death.
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Researchers have shown that transplanting stem cells derived from normal mouse blood vessels into the hearts of mice that model the pathology associated with Duchenne muscular dystrophy (DMD) prevents the decrease in heart function associated with DMD.
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Researchers at the Bellvitge Biomedical Research Institute (IDIBELL) have described a new selective target in muscle regeneration. This is the association of alpha-enolase protein and plasmin.
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A muscle relaxant called dantrolene used for treating malignant hyperthermia boosts the activity of therapies currently being developed to treat the genetic disorder Duchenne muscular dystrophy (DMD), show study findings.
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Drugs are currently being tested that show promise in treating patients with Duchenne muscular dystrophy (DMD), an inherited disease that affects about one in 3,600 boys and results in muscle degeneration and, eventually, death.
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Milo Biotechnology today announced its AAV1-FS344 has been granted Orphan Drug designation from the FDA's Office of Orphan Products Development for treatment of Becker and Duchenne muscular dystrophy. AAV1-FS344 is a gene therapy-delivered myostatin inhibitor that increases muscle strength.
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Cedars-Sinai Heart Institute researchers have found in an initial clinical trial that a drug typically prescribed for erectile dysfunction or pulmonary hypertension restores blood flow to oxygen-starved muscles in patients with a type of muscular dystrophy that affects males, typically starting in childhood or adolescence.
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Scientists have discovered that injecting a novel human protein into muscle affected by Duchenne muscular dystrophy significantly increases its size and strength, findings that could lead to a therapy akin to the use of insulin by diabetics.
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