Duchenne Muscular Dystrophy News and Research

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Duchenne muscular dystrophy (DMD) is a progressive muscle disorder that causes the loss of both muscle function and independence. DMD is perhaps the most prevalent of the muscular dystrophies and is the most common lethal genetic disorder diagnosed during childhood today. Each year, approximately 20,000 children worldwide are born with DMD (one of every 3,500 male children).

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.

9 Oct 2013

Stem cell lines are ideal research tools for designing models to understand disease progression

Scientists from King's College London have announced that 16 human embryonic stem (hES) cell lines have been approved by the US National Institutes of Health (NIH) and placed on their Stem Cell Registry, making them freely available for federally-funded research in the USA.

25 Sep 2013

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.

18 Sep 2013

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.

12 Aug 2013

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.

8 Aug 2013

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.

2 Aug 2013

Study results suggest reexamination of biological, medical importance of X chromosome

Painstaking new analysis of the genetic sequence of the X chromosome—long perceived as the "female" counterpart to the male-associated Y chromosome—reveals that large portions of the X have evolved to play a specialized role in sperm production.

24 Jul 2013

NIH awards $12.7M to research groups to explore new treatments for patients in eight disease areas

The National Institutes of Health has awarded $12.7 million to match nine academic research groups with a selection of pharmaceutical industry compounds to explore new treatments for patients in eight disease areas, including Alzheimer's disease, Duchenne muscular dystrophy and schizophrenia.

19 Jun 2013

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.

5 Jun 2013

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.

30 May 2013

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.

18 Apr 2013

Royal Holloway University supports Muscular Dystrophy Campaign

Royal Holloway University, is today giving money generated by its research into Duchenne muscular dystrophy to the Muscular Dystrophy Campaign.

27 Mar 2013

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.

21 Mar 2013

Columbia University Medical Center, NewYork-Presbyterian Hospital present research works at AAN meeting

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.

18 Mar 2013

Genetic mutation that alters PIEZO1 ion channel in RBCs contributes to familial xerocytosis

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.

11 Mar 2013

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).

7 Mar 2013

Steroid therapy impedes Duchenne’s cardiomyopathy

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.

11 Feb 2013

Researchers to develop new diagnostic tools and treatments for people with rare diseases

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.

25 Jan 2013

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.

23 Jan 2013

UC Davis professor receives supplemental funds from Parent Project Muscular Dystrophy

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).

19 Jan 2013