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Dystrophin is a rod-shaped cytoplasmic protein, and a vital part of a protein complex that connects the cytoskeleton of a muscle fiber to the surrounding extracellular matrix through the cell membrane.
Sarepta Therapeutics reports non-GAAP net loss of $29.1 million for Q4 2013

Sarepta Therapeutics reports non-GAAP net loss of $29.1 million for Q4 2013

Sarepta Therapeutics, Inc., a developer of innovative RNA-based therapeutics, today reported financial results for the three months and year ended December 31, 2013, and provided an update of recent corporate developments. [More]
Researchers demonstrate new approach to treating muscular dystrophy

Researchers demonstrate new approach to treating muscular dystrophy

Researchers at Washington University School of Medicine in St. Louis have demonstrated a new approach to treating muscular dystrophy. Mice with a form of this muscle-weakening disease showed improved strength and heart function when treated with nanoparticles loaded with rapamycin, an immunosuppressive drug recently found to improve recycling of cellular waste. [More]

Researchers overcome obstacle to using viruses to deliver therapeutic genes

Scientists in The Research Institute at Nationwide Children's Hospital have found a way to overcome one of the biggest obstacles to using viruses to deliver therapeutic genes: how to keep the immune system from neutralizing the virus before it can deliver its genetic payload. In a study published recently in Molecular Therapy, researchers found that giving subjects a treatment to temporarily rid the body of antibodies provides the virus safe passage to targeted cells, allowing it to release a corrective or replacement gene to treat disease. [More]
Three Penn Medicine researchers awarded grants for neuromuscular disease research

Three Penn Medicine researchers awarded grants for neuromuscular disease research

The Muscular Dystrophy Association has awarded research grants totaling over $1,000,000 to three Penn Medicine researchers: James Shorter, PhD, associate professor of Biochemistry and Biophysics; Hansell Stedman, MD, associate professor of Surgery; and Lee Sweeney, PhD, director of the Center for Orphan Disease Research and Therapy. [More]

VBP15 drug shows early promise for treatment of Duchenne muscular dystrophy

A preclinical study led by researchers at Children's National Medical Center has found that a new oral drug shows early promise for the treatment of Duchenne muscular dystrophy. [More]
Gene combination therapy repairs and prevents future damage of muscles caused by DMD

Gene combination therapy repairs and prevents future damage of muscles caused by DMD

New research on two promising gene therapies suggests that combining them into one treatment not only repairs muscle damage caused by Duchenne muscular dystrophy, but also prevents future injury from the muscle-wasting disease. [More]
Eteplirsen offers great hope to patients with Duchenne muscular dystrophy

Eteplirsen offers great hope to patients with Duchenne muscular dystrophy

Results from a clinical trial of eteplirsen, a drug designed to treat Duchenne muscular dystrophy, suggest that the therapy allows participants to walk farther than people treated with placebo and dramatically increases production of a protein vital to muscle growth and health. [More]
Trace substance in sugar improves muscle regeneration in mouse model with muscular dystrophy

Trace substance in sugar improves muscle regeneration in mouse model with muscular dystrophy

A trace substance in caramelized sugar, when purified and given in appropriate doses, improves muscle regeneration in a mouse model of Duchenne muscular dystrophy. The findings are published today, Aug. 1, in the journal Skeletal Muscle. [More]

Duke researchers find novel way to repair gene responsible for Duchenne muscular dystrophy

Using a novel genetic 'editing' technique, Duke University biomedical engineers have been able to repair a defect responsible for one of the most common inherited disorders, Duchenne muscular dystrophy, in cell samples from Duchenne patients. [More]

Cedars-Sinai team awarded grant to study cardiac stem cell treatment for Duchenne muscular dystrophy

Newport Beach-based nonprofit Coalition Duchenne has awarded a $150,000 grant to a Cedars-Sinai Heart Institute team investigating whether an experimental cardiac stem cell treatment could be used to treat Duchenne muscular dystrophy patients who have developed heart disease. [More]

ARMGO Pharma gets $1 million to support ARM210 preclinical work for DMD treatment

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. [More]

DART Therapeutics develops new class of therapy for Duchenne muscular dystrophy

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. [More]

Study combines induced pluripotent stem cell technology and genetic correction to treat DMD

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). [More]
Research on dystrophin gene sequence could lead to treatments for Duchenne muscular dystrophy

Research on dystrophin gene sequence could lead to treatments for Duchenne muscular dystrophy

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. [More]
Potential stem cell therapy for Duchenne’s

Potential stem cell therapy for Duchenne’s

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. [More]

Positive gene therapy results in large mammals of Duchenne muscular dystrophy

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. [More]

Stem cell transplantation prevents decrease in heart function associated with DMD

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. [More]
The association alfa-enolase/plasmin is a new selective target for treating muscular pathologies

The association alfa-enolase/plasmin is a new selective target for treating muscular pathologies

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. [More]

Dantrolene shows promise for treating DMD

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. [More]
Dantrolene may help combat Duchenne muscular dystrophy in boys

Dantrolene may help combat Duchenne muscular dystrophy in boys

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. [More]