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.

First Edition: December 7, 2012

Today's early morning highlights from the major news organizations includes articles on the talks between President Barack Obama and House Speaker John Boehner on the "fiscal cliff."

7 Dec 2012

Scientists isolate previously unknown protein in skeletal muscle

Scientists at Dana-Farber Cancer Institute have isolated a previously unknown protein in muscles that spurs their growth and increased power following resistance exercise. They suggest that artificially raising the protein's levels might someday help prevent muscle loss caused by cancer, prolonged inactivity in hospital patients, and aging.

7 Dec 2012

‘Weekend pill’ employed for muscular dystrophy

A drug used to treat erectile dysfunction may prevent functional muscle ischemia in patients with Becker muscular dystrophy, a small randomized trial shows.

6 Dec 2012

Tadalafil may offer therapeutic strategy in patients with Becker muscular dystrophy

Cedars-Sinai Heart Institute researchers have found in an initial clinical trial that a drug typically prescribed for erectile dysfunction or pulmonary hypertension restores blood flow to oxygen-starved muscles in patients with a type of muscular dystrophy that affects males, typically starting in childhood or adolescence.

29 Nov 2012

Injecting Wnt7a protein could prevent Duchenne muscular dystrophy

Scientists have discovered that injecting a novel human protein into muscle affected by Duchenne muscular dystrophy significantly increases its size and strength, findings that could lead to a therapy akin to the use of insulin by diabetics.

28 Nov 2012

New therapeutic technique to repair and rebuild muscle for degenerative muscle disorders

A study published today in BioMed Central's open access journal Skeletal Muscle reports of a new therapeutic technique to repair and rebuild muscle for sufferers of degenerative muscle disorders. The therapy brings together two existing techniques for muscle repair - cell transplantation and tissue engineering - specifically, mesoangioblast stem cells delivered via a hydrogel cell-carrier matrix.

26 Nov 2012

Myasthenia gravis therapies: an interview with Professor Daniel Drachman

Myasthenia gravis (MG) is an autoimmune disease that produces weakness and fatiguability of muscles. It affects between 1 and 7 people per 10,000, according to the best statistics.

15 Nov 2012

HCT 1026 may help stem debilitating effects of muscular dystrophy

Cedars-Sinai Heart Institute researchers have found that an experimental compound may help stem the debilitating effects of muscular dystrophy by restoring normal blood flow to muscles affected by the genetic disorder.

9 Nov 2012

University of Tübingen heads Neuromics consortium

Work has begun on Neuromics, a major project headed by Tübingen's Professor Olaf Riess; the project beat out ten other first-class applications to attain €12m euros in EU funding over five years.

8 Nov 2012

Imatinib mesylate effectively treats children with neurofibromatosis type 1 tumors

Physician-researchers at Indiana University School of Medicine have reported the first effective therapy for a class of previously untreatable and potentially life-threatening tumors often found in children.

2 Nov 2012

‘Gene for speed’ researcher awarded Ramaciotti Medal

Professor Kathryn North AM, the researcher responsible for discovering the ‘gene for speed’, has been awarded the prestigious Ramaciotti Medal for Excellence in Biomedical Research and a $50,000 grant.

16 Oct 2012

Two CUMC studies provide new insights into spinal muscular atrophy

A study by scientists from the Motor Neuron Center at Columbia University Medical Center (CUMC) suggests that spinal muscular atrophy (SMA), a genetic neuromuscular disease in infants and children, results primarily from motor circuit dysfunction, not motor neuron or muscle cell dysfunction, as is commonly thought.

12 Oct 2012

Transplanted neural stem cells able to produce new myelin in children with PMD

A Phase I clinical trial led by investigators from the University of California, San Francisco and sponsored by Stem Cells Inc., showed that neural stem cells successfully engrafted into the brains of patients and appear to have produced myelin.

11 Oct 2012

Mexiletine improves patient-reported stiffness in nondystrophic myotonia

An older medication originally approved to treat heart problems eases the symptoms of a very rare muscle disease that often leaves its sufferers stiff and in a good deal of pain, physicians and researchers report in the Oct. 3 issue of the Journal of the American Medical Association.

4 Oct 2012

New mouse model could foster ALS, Paget's and dementia breakthroughs

Efforts to treat disorders like Lou Gehrig's disease, Paget's disease, inclusion body myopathy and dementia will receive a considerable boost from a new research model created by UC Irvine scientists.

3 Oct 2012

Autoimmune disease myasthenia gravis halted in mice

Working with mice, Johns Hopkins researchers say they have developed a gene-based therapy to stop the rodent equivalent of the autoimmune disease myasthenia gravis by specifically targeting the destructive immune response the disorder triggers in the body.

1 Oct 2012

Common RNA pathway links frontotemporal lobar dementia with ALS

Two proteins previously found to contribute to ALS, also known as Lou Gehrig's disease, have divergent roles. But a new study, led by researchers at the Department of Cellular and Molecular Medicine at the University of California, San Diego School of Medicine, shows that a common pathway links them.

1 Oct 2012

Researchers solve key part of muscle regeneration mystery

Working with mice, Johns Hopkins researchers have solved a key part of a muscle regeneration mystery plaguing scientists for years, adding strong support to the theory that muscle mass can be built without a complete, fully functional supply of muscle stem cells.

28 Sep 2012

Combination of two neuroprotective therapies shows promise against Alzheimer's

The combination of two neuroprotective therapies, voluntary physical exercise, and the daily intake of melatonin has been shown to have a synergistic effect against brain deterioration in rodents with three different mutations of Alzheimer's disease.

27 Sep 2012

Researchers hold new implications for pathogenesis of myotonic dystrophy

An important breakthrough could help in the fight against myotonic dystrophy. The discovery, recently published in the prestigious scientific journal Cell, results from an international collaboration between researchers at the IRCM, the Massachusetts Institute of Technology (MIT), the University of Southern California and Illumina.

13 Sep 2012