Muscular Dystrophy News and Research

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The muscular dystrophies (MD) are a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms of MD are seen in infancy or childhood, while others may not appear until middle age or later. The disorders differ in terms of the distribution and extent of muscle weakness (some forms of MD also affect cardiac muscle), age of onset, rate of progression, and pattern of inheritance.

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

RNA interference therapy heals growth deficiency disorder in a live animal

A team of Vanderbilt researchers have demonstrated for the first time that a new type of gene therapy, called RNA interference, can heal a genetic disorder in a live animal.

20 Dec 2007

Surprising findings related to myotonic muscular dystrophy and heart protein

New research from the University of Virginia Health System shows that, in cases of Type 1 myotonic muscular dystrophy (DM1), a well known heart protein does several surprising things.

18 Dec 2007

New findings on myotonic muscular dystrophy

New research from the University of Virginia Health System shows that, in cases of Type 1 myotonic muscular dystrophy (DM1), a well known heart protein does several surprising things. DM1 is the most common form of muscular dystrophy in adults and affects approximately 40,000 adults and children in the U.S.

18 Dec 2007

Overexcited neurons not good for cell health

Neurotransmitters have consequences. They initiate events that are critical to a healthy life, giving us the ability to move, to talk, to breathe, to think. But that's if the neurotransmitters are getting it right and sending proper signals downstream to muscle cells, neurons or other cells.

18 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

Researchers identify compound that eliminates myotonia - a symptom of muscular dystrophy

Researchers at the University of Rochester Medical Center (URMC) have identified a compound that eliminates myotonia - a symptom of muscular dystrophy - in mice. The research was published online in the Journal of Clinical Investigation.

16 Nov 2007

ClC-1 helps mice get a move on

In addition to the progressive muscle weakness and degeneration common to all forms of muscular dystrophy, patients with the most common form of muscular dystrophy affecting adults, myotonic dystrophy type 1 (DM1), also experience an inability to voluntarily relax the muscles (a problem known as myotonia).

16 Nov 2007

Fat cells release a protein that aids insulin secretion from pancreatic beta cells

The body's fat cells help the pancreas do its job of secreting insulin, according to research at Washington University School of Medicine in St. Louis.

6 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

Public access policy for NIH-funded research set to become law

The U.S. Senate last night approved the FY2008 Labor, HHS, and Education Appropriations Bill (S.1710), including a provision that directs the National Institutes of Health (NIH) to strengthen its Public Access Policy by requiring rather than requesting participation by researchers.

24 Oct 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

RNA-binding protein key to understanding myotonic dystrophy type 1

Increased levels of a protein called CUGBP1 play an important role in the adult-onset form of muscular dystrophy called myotonic dystrophy type 1, said Baylor College of Medicine researchers in a report that appears in the journal Molecular Cell.

12 Oct 2007

Pennsylvania gets to grips with Fetal Alcohol Spectrum Disorder

Health officials in Pennsylvania in the U.S. are calling for more awareness of Fetal Alcohol Spectrum Disorder (FASD).

11 Sep 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

'Mighty mice' made mightier

The Johns Hopkins scientist who first showed that the absence of the protein myostatin leads to oversized muscles in mice and men has now found a second protein, follistatin, whose overproduction in mice lacking myostatin doubles the muscle-building effect.

29 Aug 2007

Innovative gene therapy research for muscular dystrophy

A major grant of approximately $10 million from the National Institutes of Health will help investigators at Columbus Children's Research Institute (CCRI) at Columbus Children's Hospital learn more about strategies to treat muscular dystrophy, potentially leading to two new gene therapy approaches.

3 Aug 2007