Dystrophin News and Research

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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.

New genetic test advances detection and diagnosis of muscular dystrophy

A new genetic test targeting the most common types of muscular dystrophy--those caused by mutations in the dystrophin gene--is far quicker with greater accuracy and sensitivity than existing tests.

28 Jun 2007

Muscle restoration in an animal model of Duchenne muscular dystrophy

Using a new type of drug that targets a specific genetic defect, researchers at the University of Pennsylvania School of Medicine, along with colleagues at PTC Therapeutics Inc. and the University of Massachusetts Medical School, have for the first time demonstrated restoration of muscle function in a mouse model of Duchenne's muscular dystrophy (DMD).

24 Apr 2007

New drug offers hope to muscular dystrophy and cystic fibrosis sufferers

Scientists in the United States say an experimental drug may be able to compensate for the genetic error responsible for some cases of Duchenne Muscular Dystrophy (DMD) and cystic fibrosis.

23 Apr 2007

Female stem cells work better

Female stem cells derived from muscle have a greater ability to regenerate skeletal muscle tissue than male cells, according to a study at Children's Hospital of Pittsburgh of UPMC.

10 Apr 2007

Revving up a crucial set of muscle genes improves muscular dystrophy symptoms in mice

Scientists at Dana-Farber Cancer Institute have shown in a laboratory study that revving up a crucial set of muscle genes counteracts the damage caused by a form of muscular dystrophy.

2 Apr 2007

Possible therapeutic target for Duchenne muscular dystrophy

In the April 1st issue of Genes & Development, Dr. Bruce Spiegelman (Dana Farber Cancer Institute) and colleagues identify a key genetic component of and possible therapeutic target for Duchenne muscular dystrophy.

2 Apr 2007

Stem cell therapy promises hope for muscular dystrophy victims

Italian scientists, by using new stem cell therapy on dogs with muscular dystrophy, have improved the dogs condition to such an extent that the dogs were able to walk and even jump again.

15 Nov 2006

New approaches to genetic disease, based on cells' own ability to correct themselves

New approaches to genetic disease, based on cells' own ability to correct themselves, will be outlined today at the annual conference of the European Society of Human Genetics in Amsterdam, The Netherlands.

8 May 2006

First gene therapy human trial in the United States for a form of muscular dystrophy under way

The clinical trial for Duchenne muscular dystrophy (DMD) tests the safety and effectiveness of a therapy that was developed over two decades by scientists at the University of North Carolina Chapel Hill's School of Medicine and the University of Pittsburgh.

16 Apr 2006

New research has implications for treating muscular dystrophy

In a surprising discovery with implications for treating muscular dystrophy, researchers at the University of Utah School of Medicine and other institutions have identified a major source of origin for two groups of adult cells that regulate muscle repair.

27 Jan 2006

New gene therapy maybe useful for treating muscular dystrophy

A new gene therapy technique that has shown promise in skin disease and hemophilia might one day be useful for treating muscular dystrophy, according to a new study by researchers at Stanford University School of Medicine.

29 Dec 2005

Unexpected link between muscular dystrophy and muscle wasting associated with cancer

A new study provides important insight into the mechanisms of a muscle wasting disorder that interferes with treatment for cancer and has a negative impact on patient survival.

5 Dec 2005

Better understanding of cancer cachexia

New research shows that a wasting condition responsible for nearly a third of all cancer deaths involves the loss of an essential muscle protein that is also lost in people with muscular dystrophy.

15 Nov 2005

Early diagnosis and treatment of heart disease may lead to longer life in Duchenne and Becker muscular dystrophy patients

Cardiac disease, particularly dilated cardiomyopathy and heart failure, is the major cause of mortality in patients with muscular dystrophy and is present in most boys with Duchenne muscular dystrophy and approximately 70 percent of those with Becker muscular dystrophy. These are the two common forms of muscular dystrophy caused by defects in a gene called dystrophin.

30 Oct 2005

Bone marrow-derived cells fail to meet high expectations says Bonn study

Adult stem cells taken from bone marrow are the "shooting stars" of their field. Many research scientists have been speculating that the cells might be able to pass through the blood into diseased organs and replace defective tissue. Such cells are seen as the potential key to the treatment of certain muscle diseases.

3 Aug 2005

Chemical sealant - poloxamer 188 can repair damage to cardiac muscle cell membrane

A common chemical used in the manufacturing and pharmaceutical industries can repair damage to cardiac muscle cell membranes and prevent heart failure in mice with the genetic mutation that causes Duchenne muscular dystrophy, according to scientists at the University of Michigan Medical School.

18 Jul 2005

Corticosteroids can be beneficial in the treatment of Duchenne muscular dystrophy

Corticosteroids can be beneficial in the treatment of Duchenne muscular dystrophy and can be offered as a treatment option, according to the American Academy of Neurology and the Child Neurology Society in a new practice guideline published in the January 11 issue of Neurology, the scientific journal of the American Academy of Neurology.

10 Jan 2005

Novel strategy for stimulating the production of utrophin

Researchers at the University of Pennsylvania School of Medicine report a novel strategy for stimulating the production of utrophin – an important muscle protein in young mice – for muscular dystrophy therapy.

21 Sep 2004

Proof of principle that it is possible to deliver new genes body-wide to all the muscles of an adult animal

MDA-supported researchers at the University of Washington-Seattle have delivered the gene for the dystrophin protein to all voluntary muscles with a single intravenous injection in mice with Duchenne muscular dystrophy (DMD).

29 Jul 2004

$1.6 million to develop gene therapy strategies for Duchenne muscular dystrophy, a fatal, childhood-onset disease

The Muscular Dystrophy Association has announced it has awarded $1.6 million to Asklepios, a biotechnology company closely allied with the University of North Carolina at Chapel Hill, to develop gene therapy strategies for Duchenne muscular dystrophy (DMD), a fatal, childhood-onset disease.

23 Jul 2004