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.

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

Dysfunctional RyRs play a role in stress-induced cognitive dysfunction

ARMGO Pharma, Inc., a biopharmaceutical company developing innovative small molecule drugs known as "Rycals™," which act on the ryanodine receptor calcium-release channel (RyR), announced that a study published today in the prestigious scientific journal Cell reveals the underlying role of calcium leak through the RyR as an important contributor to stress-induced cognitive dysfunction.

1 Sep 2012

S107 drug can prevent learning and memory deficits associated with stress-related disorders

Columbia University Medical Center (CUMC) researchers have identified a potential medical treatment for the cognitive effects of stress-related disorders, including post-traumatic stress disorder (PTSD).

31 Aug 2012

Grb10 plays a crucial role in increasing muscle mass during development

Scientists have moved closer toward helping people grow big, strong muscles without needing to hit the weight room. Australian researchers have found that by blocking the function of a protein called Grb10 while mice were in the womb, they were considerably stronger and more muscular than their normal counterparts.

31 Aug 2012

Microtubule stiffening worsens skeletal muscle degeneration in Duchenne muscular dystrophy

University of Maryland (UM) researchers and collaborators report in the journal Science Signaling that skeletal muscle degeneration in Duchenne muscular dystrophy (DMD) is worsened by stiffening of the microtubule cytoskeleton that provides structure inside muscle cells.

23 Aug 2012

Laminin-111 protein therapy could treat Duchenne muscular dystrophy

A novel treatment in development at the University of Nevada School of Medicine for the most common form of muscular dystrophy is advancing towards human trials with a $308,000 boost from the Muscular Dystrophy Association.

23 Aug 2012

Trial to test efficacy of NeuRx Diaphragm Pacing System in people with Lou Gehrig's disease

The Muscular Dystrophy Association (MDA) and The ALS Association today announced a joint commitment to provide funding for a phase 2 clinical trial that will test efficacy of the NeuRx Diaphragm Pacing System (DPS) in people with amyotrophic lateral sclerosis (ALS).

15 Aug 2012

Antisense therapy shows promise in treating toxoplasmosis

A targeted approach to treating toxoplasmosis, a parasitic disease, shows early promise in test-tube and animal studies, where it prevented the parasites from making selected proteins. When tested in newly infected mice, it reduced the number of viable parasites by more than 90 percent, researchers from the University of Chicago Medicine report in the Proceedings of the National Academy of Sciences.

14 Aug 2012

Molecular missteps disrupt brain function in adult-onset muscular dystrophy

The molecular missteps that disrupt brain function in the most common form of adult-onset muscular dystrophy have been revealed in a new study published by Cell Press. Myotonic dystrophy is marked by progressive muscle wasting and weakness, as well as excessive daytime sleepiness, memory problems, and mental retardation.

10 Aug 2012

Researchers identify myostatin as potential therapeutic target for muscular disorders

The study of muscular system protein myostatin has been of great interest to researchers as a potential therapeutic target for people with muscular disorders. Although much is known about how myostatin affects muscle growth, there has been disagreement about what types of muscle cells it acts upon. New research from a team including Carnegie's Chen-Ming Fan and Christoph Lepper narrows down the field to one likely type of cell.

7 Aug 2012

Montefiore launches first fellowship program in Women's Physical Medicine and Rehabilitation

Montefiore Medical Center has launched the nation's first fellowship program in Women's Physical Medicine and Rehabilitation (WPM&R), and established a new clinical program in this specialty.

6 Aug 2012

Myotonia less important to patients with myotonic dystrophy than other symptoms

Complex, multi-system diseases like myotonic dystrophy - the most common adult form of muscular dystrophy - require physicians and patients to identify which symptoms impact quality of life and, consequently, what treatments should take priority. However, a new study out this month in the journal Neurology reveals that there is often a disconnect between the two groups over which symptoms are more important, a phenomenon that not only impacts care but also the direction of research into new therapies.

26 Jul 2012

Rapamycin improves function and extends survival in mice with DCM and rare muscular dystrophies

Rapamycin, an immunosuppressant drug used in a variety of disease indications and under study in aging research labs around the world, improved function and extended survival in mice suffering from a genetic mutation which leads to dilated cardiomyopathy (DCM) and rare muscular dystrophies in humans.

26 Jul 2012

Researchers discover new cause of congenital myopathy

University of Michigan researchers have discovered a new cause of congenital myopathy: a mutation in a previously uncharacterized gene, according to research published this month in the American Journal of Human Genetics.

26 Jul 2012

PTC Therapeutics completes $30M financing

PTC Therapeutics, Inc. (PTC) today announced the completion of a $30 million financing. The round included a number of long-term investors such as Credit Suisse First Boston Equity Partners, HBM BioVentures, Vulcan Ventures, Celgene, Delphi Ventures, The Column Group, Novo A/S, and other existing investors.

23 Jul 2012

Venom protein could treat Duchenne muscular dystrophy

While Spider-Man is capturing the imagination of theatergoers, real-life spider men in Upstate New York are working intently to save a young boy's life. It all began in 2009, when Jeff Harvey, a stockbroker from the Buffalo suburbs, discovered that his grandson, JB, had Duchenne muscular dystrophy. The disease is fatal. It strikes only boys, causing their muscles to waste away.

17 Jul 2012

UMMS scientists identify new genetic mutation that causes familial ALS

A new genetic mutation that causes familial amyotrophic lateral sclerosis (ALS), a fatal neurological disorder also known as Lou Gehrig's Disease, has been identified by a team of scientists led by researchers at the University of Massachusetts Medical School (UMMS).

16 Jul 2012

New eye-tracking device for amputees

Millions of people suffering from Multiple Sclerosis, Parkinson's, muscular dystrophy, spinal cord injuries or amputees could soon interact with their computers and surroundings using just their eyes, thanks to a new device that costs less than £40.

16 Jul 2012