Muscular Dystrophy News and Research RSS Feed - Muscular Dystrophy News and Research

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.
CUMC researchers uncover new details of intracellular channel that controls skeletal muscle

CUMC researchers uncover new details of intracellular channel that controls skeletal muscle

Using high-resolution electron microscopy, Columbia University Medical Center researchers have uncovered new details of the structure and function of an intracellular channel that controls the contraction of skeletal muscle. [More]
Sarepta Therapeutics wins accelerated approval from FDA for Duchenne muscular dystrophy drug

Sarepta Therapeutics wins accelerated approval from FDA for Duchenne muscular dystrophy drug

The U.S. Food and Drug Administration today approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD). [More]
New research could pave way for safe, effective treatment for muscular dystrophy

New research could pave way for safe, effective treatment for muscular dystrophy

New research has shown that the corticosteroid deflazacort is a safe and effective treatment for Duchenne muscular dystrophy. The findings, which appear this month in the journal Neurology, could pave the way for first U.S.-approved treatment for the disease. [More]
Christopher & Dana Reeve Foundation awards Quality of Life grants to 79 nonprofit organizations

Christopher & Dana Reeve Foundation awards Quality of Life grants to 79 nonprofit organizations

The Christopher & Dana Reeve Foundation, a national nonprofit dedicated to improving quality of life for individuals living with paralysis, announced it has awarded $575,704 in Quality of Life grants to 79 nonprofit organizations nationwide. [More]
MDI Biological Laboratory researchers receive patent for novel heart disease drug

MDI Biological Laboratory researchers receive patent for novel heart disease drug

The U.S. Patent and Trademark Office has announced that it will grant a patent to MDI Biological Laboratory scientists Voot P. Yin, Ph.D., and Kevin Strange, Ph.D., and their collaborator Michael Zasloff, M.D., Ph.D., for use of the small molecule MSI-1436 to stimulate the repair and regeneration of heart tissue damaged by injuries such as a heart attack. [More]
Penn study sheds light on role of essential compound in maintaining optimal muscle function

Penn study sheds light on role of essential compound in maintaining optimal muscle function

Maintaining proper levels of an essential helper molecule is crucial for optimal muscle function, according to a study led by Joseph Baur, PhD, an assistant professor of Physiology in the Perelman School of Medicine at the University of Pennsylvania. [More]
New gene therapeutic approach could save people suffering from muscle wasting disease

New gene therapeutic approach could save people suffering from muscle wasting disease

A discovery by Washington State University scientist Dan Rodgers and collaborator Paul Gregorevic could save millions of people suffering from muscle wasting disease. [More]
Key gene controls ability of adult stem cells to regenerate muscle after injury, study finds

Key gene controls ability of adult stem cells to regenerate muscle after injury, study finds

A key gene enables the repair of injured muscle throughout life. This is the finding of a study in mice led by researchers at NYU Langone Medical Center and the University of Colorado at Boulder, and published online July 21 in Cell Reports. [More]
Protein present on stem cell surface can boost growth of damaged muscle tissue

Protein present on stem cell surface can boost growth of damaged muscle tissue

Johns Hopkins University biologists have found that a protein that plays a key role in the lives of stem cells can bolster the growth of damaged muscle tissue, a step that could potentially contribute to treatments for muscle degeneration caused by old age and diseases such as muscular dystrophy. [More]
Scientists explore black box of genome biology

Scientists explore black box of genome biology

Scientists at Florida State University, Baylor College of Medicine and the Broad Institute of Harvard and MIT have broken ground in a little-understood area of human genetics. [More]
Researchers study how skin stem cells respond to forces

Researchers study how skin stem cells respond to forces

All cells share the same genetic code, no matter if they are skin or brain cells. However, these cells are exposed to very different types of mechanical environments and mechanical stresses. [More]
AAN, ABF and MDA offer new two-year clinical training fellowship in muscular dystrophy

AAN, ABF and MDA offer new two-year clinical training fellowship in muscular dystrophy

The American Academy of Neurology, the American Brain Foundation and the Muscular Dystrophy Association have announced a new Clinical Research Training Fellowship in muscular dystrophy for 2017. [More]
New research reveals b1-integrin protein as promising therapeutic target for muscle aging

New research reveals b1-integrin protein as promising therapeutic target for muscle aging

As we age, the function and regenerative abilities of skeletal muscles deteriorate, which means it is difficult for the elderly to recover from injury or surgery. New work from Carnegie's Michelle Rozo, Liangji Li, and Chen-Ming Fan demonstrates that a protein called b1-integrin is crucial for muscle regeneration. [More]
LIF-treated muscle stem cells show promise in treatment of muscular dystrophy

LIF-treated muscle stem cells show promise in treatment of muscular dystrophy

Satellite cells are stem cells found in skeletal muscles. While transplantation of such muscle stem cells can be a potent therapy for degenerative muscle diseases such as Duchenne muscular dystrophy, these cells tend to lose their transplantation efficiency when cultured in vitro. [More]
Re-engineering cardiac scarring process could improve clinical outcomes for heart disease patients

Re-engineering cardiac scarring process could improve clinical outcomes for heart disease patients

Literal heartbreak, from illness or injury, triggers the body's natural healing mechanisms. [More]
Neurofibromatosis causes benign tumor formation in normal nervous tissue

Neurofibromatosis causes benign tumor formation in normal nervous tissue

Although neurofibromatosis (NF) is not commonly discussed, it affects more than 2 million people worldwide. [More]
Researchers clarify cause of heart arrhythmia in myotonic dystrophy

Researchers clarify cause of heart arrhythmia in myotonic dystrophy

An international joint research group found that the cause of heart arrhythmia in myotonic dystrophy was RNA abnormalities in the sodium channel in the heart, clarifying the symptom's mechanism. This finding will be helpful in prevention and early intervention of death in this disease, leading to the development of new treatment. [More]

Enzyme protein neutrophil elastase may be key contributor to development of muscular dystrophy

Scientists at the University of Liverpool have discovered that muscle cells affected by muscular dystrophy contain high levels of an enzyme that impairs muscle repair. This finding provides a new target for potential drug treatments for the disease, which currently has no cure. [More]
Study reveals gpihbp1 gene could be novel target for treatment of muscular dystrophy

Study reveals gpihbp1 gene could be novel target for treatment of muscular dystrophy

The average healthy man is 54 percent muscle by mass, but people with muscular dystrophy, an incurable, genetic condition, have almost no muscle at terminal stages of the disease. [More]
Research sheds light on how subtle genetic differences in DMD patients produce variation in symptoms

Research sheds light on how subtle genetic differences in DMD patients produce variation in symptoms

Johns Hopkins researchers report they have inadvertently found a way to make human muscle cells bearing genetic mutations from people with Duchenne muscular dystrophy (DMD). [More]
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