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

Duchenne muscular dystrophy (DMD) is a progressive muscle disorder that causes the loss of both muscle function and independence. DMD is perhaps the most prevalent of the muscular dystrophies and is the most common lethal genetic disorder diagnosed during childhood today. Each year, approximately 20,000 children worldwide are born with DMD (one of every 3,500 male children).
Cedars-Sinai receives $7.3 million grant to test safety of novel cell-based therapy in treating PAH

Cedars-Sinai receives $7.3 million grant to test safety of novel cell-based therapy in treating PAH

Researchers from the Cedars-Sinai Heart Institute and the Cedars-Sinai Department of Medicine are expanding their ongoing evaluation of a novel cell-based therapeutic candidate into the area of pulmonary arterial hypertension (PAH). [More]
New drug treatment can override genetic fault that causes choroideremia

New drug treatment can override genetic fault that causes choroideremia

Researchers with funding from Fight for Sight have demonstrated that a new drug treatment for cystic fibrosis and Duchenne muscular dystrophy can override a genetic fault that causes choroideremia – a severe blinding disorder. [More]
FDA approval of controversial drug for Duchenne muscular dystrophy raises concern and hope

FDA approval of controversial drug for Duchenne muscular dystrophy raises concern and hope

In September, the Food and Drug Administration approved Exondys, a controversial treatment for Duchenne muscular dystrophy based on tenuous data from just 12 patients. [More]
Fight for Sight, Thomas Pocklington collaborate to fund new neuro-ophthalmology research

Fight for Sight, Thomas Pocklington collaborate to fund new neuro-ophthalmology research

Retinal activity in autism, ADHD and Duchenne muscular dystrophy [More]
Immune system plays important role in Duchenne muscular dystrophy, research reveals

Immune system plays important role in Duchenne muscular dystrophy, research reveals

A new paper, co-written by faculty at Binghamton University, State University of New York, increases the understanding of Duchenne muscular dystrophy (DMD)—one of the most common lethal genetic disorders—and points to potential therapeutic approaches. [More]
First comprehensive center opened in Tri-State area to mark new era in care of DMD patients

First comprehensive center opened in Tri-State area to mark new era in care of DMD patients

Nearly 1,000 boys in the New York Tri-State area have been diagnosed with Duchenne Muscular Dystrophy (DMD) and, until now, had to travel out of the state for comprehensive care. [More]
Department of Defense funds cardiac cell therapy trial for heart failure patients

Department of Defense funds cardiac cell therapy trial for heart failure patients

Building on the results of a recent Cedars-Sinai Heart Institute study published just six months ago, the Department of Defense has awarded a $10 million grant to fund a cardiac cell therapy trial for patients diagnosed with a common but difficult-to-treat form of heart failure. [More]
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]
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]
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]
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]
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]
Enzyme protein neutrophil elastase may be key contributor to development of muscular dystrophy

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]
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]
Removing immunomodulatory protein improves symptoms of muscular dystrophy in mice

Removing immunomodulatory protein improves symptoms of muscular dystrophy in mice

Removing an immunomodulatory protein called osteopontin improves the symptoms of mice with muscular dystrophy by changing the type of macrophages acting on damaged muscle tissue, according to a paper published in The Journal of Cell Biology. [More]
Researchers investigate biochemical, physiological characteristics of facial and extraocular muscles

Researchers investigate biochemical, physiological characteristics of facial and extraocular muscles

In a new study, a research team at Basel University Hospital in Switzerland investigates the biochemical and physiological characteristics of orbicularis oculi, a group of facial muscles that control the eyelids and are selectively spared or involved in different neuromuscular disorders. What they found also helps to explain why another set of muscles—the extraocular muscles that control the movement of the eye—are not affected by Duchenne muscular dystrophy, congenital muscular dystrophy, and aging. [More]
Long-term exercise may benefit Spinal Muscular Atrophy patients

Long-term exercise may benefit Spinal Muscular Atrophy patients

Long-term exercise appears to be beneficial for Spinal Muscular Atrophy (SMA) like mice, suggesting a potential of active physiotherapy for patient care; according to a study published today in the Journal of Physiology. [More]
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