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.

New targeted enzyme replacement therapy can treat X-linked myotubular myopathy

A collaborative research team including a Medical College of Wisconsin (MCW) pediatric neuropathologist successfully mitigated some of the effects of a muscular disease by using a new targeted enzyme replacement therapy strategy from 4s3 Bioscience.

23 Jan 2013

Research on dystrophin gene sequence could lead to treatments for Duchenne muscular dystrophy

Muscular dystrophy is caused by the largest human gene, a complex chemical leviathan that has confounded scientists for decades. Research conducted at the University of Missouri and described this month in the Proceedings of the National Academy of Sciences has identified significant sections of the gene that could provide hope to young patients and families.

23 Jan 2013

UC Davis professor receives supplemental funds from Parent Project Muscular Dystrophy

Parent Project Muscular Dystrophy (PPMD) has awarded Dr. Craig McDonald of the University of California, Davis (UC Davis) $175,000 in supplemental funds to expand his ongoing study through the 20 CINRG (Cooperative International Neuromuscular Research Group) centers to better understand the progression of Duchenne muscular dystrophy (Duchenne) and determine the impact of the Duchenne standards of care established by the Centers for Disease Control (CDC).

19 Jan 2013

Potential stem cell therapy for Duchenne’s

Researchers have found that injecting aorta-derived stem cells into the hearts of dystrophin-deficient mice prevents the onset of dilated cardiomyopathy, raising hopes of a potential new treatment approach to prevent or reverse the condition in human Duchenne muscular dystrophy.

17 Jan 2013

Positive gene therapy results in large mammals of Duchenne muscular dystrophy

Usually, results from a new study help scientists inch their way toward an answer whether they are battling a health problem or are on the verge of a technological breakthrough. Once in a while, those results give them a giant leap forward. In a preliminary study in a canine model of Duchenne muscular dystrophy (DMD), University of Missouri scientists showed exactly such a leap using gene therapy to treat muscular dystrophy.

17 Jan 2013

Tamoxifen can counteract some pathologic features in mouse model of DMD

A new study has found that tamoxifen, a well-known breast cancer drug, can counteract some pathologic features in a mouse model of Duchenne muscular dystrophy (DMD). At present, no treatment is known to produce long-term improvement of the symptoms in boys with DMD, a debilitating muscular disorder that is characterized by progressive muscle wasting, respiratory and cardiac impairments, paralysis, and premature death.

16 Jan 2013

Stem cell transplantation prevents decrease in heart function associated with DMD

Researchers have shown that transplanting stem cells derived from normal mouse blood vessels into the hearts of mice that model the pathology associated with Duchenne muscular dystrophy (DMD) prevents the decrease in heart function associated with DMD.

16 Jan 2013

Pfizer, Repligen sign licensing deal to advance spinal muscular atrophy program

Repligen Corporation announced today that it has entered into an exclusive worldwide licensing agreement with Pfizer Inc. to advance Repligen's spinal muscular atrophy (SMA) program, originally in-licensed from Families of SMA (FSMA).

3 Jan 2013

Researchers develop unique approach to calpain inhibition

Calpain, a calcium-regulated enzyme, is essential to a host of cellular processes, but can cause severe problems in its overactivated state. It has been implicated as a factor in muscular dystrophy, AIDS, Alzheimer's disease, multiple sclerosis, and cancer. As such, finding and exploiting calpain inhibitors is an important area of research.

28 Dec 2012

New technique may prevent inheritance of mitochondrial diseases in children

A joint team of scientists from The New York Stem Cell Foundation (NYSCF) Laboratory and Columbia University Medical Center (CUMC) has developed a technique that may prevent the inheritance of mitochondrial diseases in children.

21 Dec 2012

Link made between muscular dystrophy and defective nerve wiring

Working with mice, Johns Hopkins scientists have discovered that a particular protein helps nerve cells extend themselves along the spinal cord during mammalian development. Their results shed light on the subset of muscular dystrophies that result from mutations in the gene that holds the code for the protein, called dystroglycan, and also show how the nerve and muscle failings of the degenerative diseases are related.

20 Dec 2012

AAV-based gene therapy cocktail may help extend lives of children with Canavan disease

Results of a clinical trial that began in 2001 show that a gene therapy cocktail conveyed into the brain by a molecular special delivery vehicle may help extend the lives of children with Canavan disease, a rare and fatal neurodegenerative disorder.

20 Dec 2012

The association alfa-enolase/plasmin is a new selective target for treating muscular pathologies

Researchers at the Bellvitge Biomedical Research Institute (IDIBELL) have described a new selective target in muscle regeneration. This is the association of alpha-enolase protein and plasmin.

19 Dec 2012

Dantrolene shows promise for treating DMD

A muscle relaxant called dantrolene used for treating malignant hyperthermia boosts the activity of therapies currently being developed to treat the genetic disorder Duchenne muscular dystrophy (DMD), show study findings.

17 Dec 2012

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