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.

Dantrolene may help combat Duchenne muscular dystrophy in boys

Drugs are currently being tested that show promise in treating patients with Duchenne muscular dystrophy (DMD), an inherited disease that affects about one in 3,600 boys and results in muscle degeneration and, eventually, death.

13 Dec 2012

FDA grants Orphan Drug designation to Milo Biotechnology's AAV1-FS344 inhibitor

Milo Biotechnology today announced its AAV1-FS344 has been granted Orphan Drug designation from the FDA's Office of Orphan Products Development for treatment of Becker and Duchenne muscular dystrophy. AAV1-FS344 is a gene therapy-delivered myostatin inhibitor that increases muscle strength.

12 Dec 2012

GSK, Fred Hutch to develop therapeutics against facioscapulohumeral muscular dystrophy

Fred Hutchinson Cancer Research Center and GlaxoSmithKline PLC (GSK) today announced a partnership to develop therapeutics to treat an inherited form of muscular dystrophy.

11 Dec 2012

Access4Kids benefits children with fine motor impairments

Imagine not being able to touch a touch-screen device. Tablets and smartphones-with all their educational, entertaining and social benefits-would be useless. Researchers at Georgia Tech are trying to open the world of tablets to children whose limited mobility makes it difficult for them to perform the common pinch and swipe gestures required to control the devices.

11 Dec 2012

FoxP3 gene expression identifies ALS disease progression in 80% of patients

The debilitating symptoms of amyotrophic lateral sclerosis, or ALS, appear to be increased by a lack of inflammation-reducing T cells, report scientists from the Methodist Neurological Institute in an upcoming print issue of The EMBO Molecular Medicine Journal.

10 Dec 2012

Roundup: Ga. 'provider fee' defeat could mean $430M less for Medicaid; Minn. health programs in line for $37M cut over fiscal cliff; Calif. stem cell board criticized

Just weeks before the 2013 General Assembly convenes, the state's provider fee, called a "bed tax" by some, remains a high-profile wild card -- one that could deal Georgia's hospital industry a crippling blow. If the fee is not renewed -- and anti-tax forces will oppose it -- the Medicaid system may lose $430 million-plus that the assessment now generates. That loss would swell the state's Medicaid financial shortfall, which is already about $400 million (Miller, 12/6).

7 Dec 2012

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