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).

New type of gene manipulation therapy offers hope for Duchenne muscular dystrophy sufferers

A medical research team at Carolinas Medical Center in Charlotte headed by Qi Long Lu, M.D., Ph.D., has made a discovery that holds promise to restore muscle function in patients afflicted by Duchenne muscular dystrophy (DMD).

21 Sep 2008

Proof-of-concept that purified muscle stem cells can be used in therapy

By injecting purified stem cells isolated from adult skeletal muscle, researchers have shown they can restore healthy muscle and improve muscle function in mice with a form of muscular dystrophy. Those muscle-building stem cells were derived from a larger pool of so-called satellite cells that normally associate with mature muscle fibers and play a role in muscle growth and repair.

10 Jul 2008

Sildenafil could become a treatment for Duchenne muscular dystrophy

A new study appears to have found another use for the drug Viagra other than for treating problems such as erectile dysfunction.

15 May 2008

Discovery of gene mutation which causes epilepsy and mental retardation - but only in women

An Australian and British research team have discovered a gene mutation that causes epilepsy and mental retardation but only in women.

12 May 2008

Investigational drug Debio-025 tested for preventing muscle fiber death in muscular dystrophy

An investigational antiviral drug currently undergoing human trials in Europe for treating Hepatitis C infections may have potential to reduce muscle cell damage in Duchenne and other forms of muscular dystrophy (MD).

17 Mar 2008

Long-term muscle improvements shown in gene therapy study in mice

Injecting a gene responsible for making a specific protein into a mouse that's used as a model for muscular dystrophy can lead to long-term improvements in the animal's muscle size and strength, a new study shows.

14 Mar 2008

Potential new gene therapy strategy for muscle-wasting diseases

Investigators in The Research Institute at Nationwide Children's Hospital have identified the role of a protein that could potentially lead to new clinical treatments to combat musculoskeletal diseases, including Duchenne muscular dystrophy (DMD).

11 Mar 2008

Long-term muscle improvements shown in gene therapy study in mice

11 Mar 2008

Experimental drug shows promise for cystic fibrosis

An experimental drug that has proven effective in treating muscular dystrophy also works for cystic fibrosis, according to researchers at the University of Alabama at Birmingham (UAB).

5 Feb 2008

Myostatin inhibitors may make injuries more likely

Block the action of a protein that normally regulates muscle mass, and watch your muscles grow.

23 Jan 2008

Stem cell therapy restores muscle function in mice with muscular dystrophy

Scientists in Texas have been able to restore muscle function in mice with muscular dystrophy by using embryonic stem cells.

21 Jan 2008

Stem-cell transplantation improves muscles in MD animal model

Using embryonic stem cells from mice, UT Southwestern Medical Center researchers have prompted the growth of healthy - and more importantly, functioning - muscle cells in mice afflicted with a human model of Duchenne muscular dystrophy.

21 Jan 2008

Personalized medicine will alter treatment of genetic disorders

One of the nation's pre-eminent genetic researchers, Eric Hoffman, PhD, of Children's Research Institute at Children's National Medical Center, predicts that in relatively short order, medicine's next innovation--individualized molecular therapies--will have the unprecedented ability to treat muscular dystrophies, and other disorders.

28 Dec 2007

Reprogrammed human adult stem cells rescue diseased muscle in mice

Scientists report that adult stem cells isolated from humans with muscular dystrophy can be genetically corrected and used to induce functional improvement when transplanted into a mouse model of the disease.

13 Dec 2007

Australian scientist awarded $1 million by Pfizer Australia to develop gene therapies for muscle-related diseases

An Australian scientist has been awarded a $1 million research grant to develop innovative gene therapy tools for muscle-related diseases.

27 Nov 2007

Gene, stem cell therapy only needs to be 50 percent effective to create a healthy heart

Heart disease is the leading cause of death in the United States and greatly affects the quality and length of life for individuals with specific forms of muscular dystrophy.

1 Nov 2007

Oral medication restores missing protein in boys with muscular dystrophy

PTC124, an oral medication that changes the way muscle cells interpret genetic information, holds promise as a treatment for some patients with Duchenne muscular dystrophy (DMD), the Muscular Dystrophy Association ( http://www.mda.org/ ) announced today.

19 Oct 2007

Massive microRNA scan uncovers leads to treating muscle degeneration

Researchers have discovered the first microRNAs - tiny bits of code that regulate gene activity - linked to each of 10 major degenerative muscular disorders, opening doors to new treatments and a better biological understanding of these debilitating, poorly understood, often untreatable diseases.

18 Oct 2007

Children with neuromuscular diseases at risk for decreased bone density

Children with neuromuscular diseases such as muscular dystrophy, may be at risk for suboptimal bone density due to low levels of vitamin D.

18 Oct 2007

Myoendothelial cells identified as new human source of stem cells with potential to repair muscle

For the first time, scientists at Children's Hospital of Pittsburgh of UPMC have discovered a unique population of adult stem cells derived from human muscle that could be used to treat muscle injuries and diseases such as heart attack and muscular dystrophy.

5 Sep 2007