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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).
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]
New approach could be used to treat Duchenne muscular dystrophy

New approach could be used to treat Duchenne muscular dystrophy

Scientists at the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research and Center for Duchenne Muscular Dystrophy at UCLA have developed a new approach that could eventually be used to treat Duchenne muscular dystrophy. [More]
Duchenne muscular dystrophy: direct effect on muscle stem cells? An interview with Dr Rudnicki

Duchenne muscular dystrophy: direct effect on muscle stem cells? An interview with Dr Rudnicki

For twenty years, it has been understood that dystrophin is expressed in differentiated muscle fibers where it is part of a protein complex that crosses the membrane and connects the extracellular matrix to the actin network inside the cell to provide structural integrity. [More]

New robotic arm could support daily activities of patients with Duchenne Muscular Dystrophy

Researchers from the University of Twente's MIRA research centre, together with the VUmc, TU Delft and the Radboud umc, have developed the A-Gear: a robotic arm that can support the daily activities of people suffering the muscular disease Duchenne Muscular Dystrophy. [More]
Leukaemia drug could slow progression of Duchenne muscular dystrophy

Leukaemia drug could slow progression of Duchenne muscular dystrophy

A drug commonly used to treat leukaemia is showing potential as a treatment that could slow the progression of the muscle-wasting condition, Duchenne muscular dystrophy. [More]
UT Southwestern scientists use new gene-editing technique to prevent progression of DMD in young mice

UT Southwestern scientists use new gene-editing technique to prevent progression of DMD in young mice

Using a new gene-editing technique, a team of scientists from UT Southwestern Medical Center stopped progression of Duchenne muscular dystrophy (DMD) in young mice. [More]
Researchers successfully use CRISPR to treat adult mouse model of Duchenne muscular dystrophy

Researchers successfully use CRISPR to treat adult mouse model of Duchenne muscular dystrophy

Researchers have used CRISPR to treat an adult mouse model of Duchenne muscular dystrophy. This marks the first time that CRISPR has successfully treated a genetic disease inside a fully developed living mammal with a strategy that has the potential to be translated to human therapy. [More]
UofL researchers discover mechanism involved in skeletal muscle repair

UofL researchers discover mechanism involved in skeletal muscle repair

Researchers at the University of Louisville have discovered a mechanism involved in skeletal muscle repair that may enable clinicians to boost the effectiveness of adult stem cell therapies for diseases such as muscular dystrophy. [More]
Could statins treat muscular dystrophy? An interview with Dr. Nick Whitehead and Dr Stan Froehner

Could statins treat muscular dystrophy? An interview with Dr. Nick Whitehead and Dr Stan Froehner

In addition to their well established cholesterol lowering benefits, statins also have potent anti-inflammatory, anti-fibrotic and antioxidant effects, which continue to be identified in a wide range of diseases. [More]
Duchenne muscular dystrophy could directly affect muscle stem cells

Duchenne muscular dystrophy could directly affect muscle stem cells

A new study from The Ottawa Hospital and the University of Ottawa is poised to completely change our understanding of Duchenne muscular dystrophy and pave the way for far more effective treatments. [More]
Simple, low risk blood test can detect foetal blood group and genetic conditions in unborn babies

Simple, low risk blood test can detect foetal blood group and genetic conditions in unborn babies

Research into a simple, accurate and low risk blood test that can detect foetal blood group, sex, and genetic conditions in unborn babies has been published in the international scientific journal, Clinical Chemistry. [More]
MU researchers successfully treat dogs with DMD, plan for human clinical trials

MU researchers successfully treat dogs with DMD, plan for human clinical trials

Muscular dystrophy, which affects approximately 250,000 people in the U.S., occurs when damaged muscle tissue is replaced with fibrous, fatty or bony tissue and loses function. For years, scientists have searched for a way to successfully treat the most common form of the disease, Duchenne Muscular Dystrophy (DMD), which primarily affects boys. Now, a team of University of Missouri researchers have successfully treated dogs with DMD and say that human clinical trials are being planned in the next few years. [More]
New hope for patients with Duchenne muscular dystrophy

New hope for patients with Duchenne muscular dystrophy

New hope for Duchenne muscular dystrophy (DMD) patients. A mouse genetic study in PLoS Medicine reports that targeting the P2RX7 gene, a purinoreceptor, may halt the progression of DMD. [More]
Researchers identify new therapeutic target for Duchenne muscular dystrophy

Researchers identify new therapeutic target for Duchenne muscular dystrophy

Scientists have found what could one day lead to a promising treatment for those with a crippling disease, Duchenne muscular dystrophy. [More]
Study: RNA editing technique shows promise in treating rare, severe form of muscular dystrophy

Study: RNA editing technique shows promise in treating rare, severe form of muscular dystrophy

An RNA editing technique called "exon skipping" has shown preliminary success in treating a rare and severe form of muscular dystrophy that currently has no treatment, based on a new study from Northwestern Medicine and the University of Chicago. Children with the disease lose significant muscle strength early in life. [More]
Tarix Orphan's TXA127 granted FDA Fast Track Designation for treatment of DMD patients

Tarix Orphan's TXA127 granted FDA Fast Track Designation for treatment of DMD patients

Tarix Orphan LLC, a privately held biopharmaceutical company focused on the treatment of rare neuromuscular disorders and connective tissue diseases, today announced that the U.S. Food and Drug Administration has granted Fast Track Designation to TXA127 (angiotensin 1-7) to reduce skeletal muscle damage and fibrosis and thereby improve muscle strength in Duchenne Muscular Dystrophy (DMD) patients. [More]
AMO Pharma raises $25 million in private equity financing

AMO Pharma raises $25 million in private equity financing

AMO Pharma Limited, a privately held biopharmaceutical company focusing on debilitating diseases with limited or no treatment options, announced today that the company has raised $25 million in private equity financing from Woodford Investment Management. [More]
New gene therapy approach to treating DMD reduces symptoms, extends life span in mouse model

New gene therapy approach to treating DMD reduces symptoms, extends life span in mouse model

A gene therapy approach to treating the progressive muscle wasting disorder Duchenne muscular dystrophy (DMD) that does not replace the mutated DMD gene but instead delivers the gene for ITGA7, a protein in skeletal muscle, led to reduced symptoms and significantly extended life span in a mouse model of severe DMD. Over-expression of ITGA7 did not elicit an immune reaction, further supporting its potential as a novel treatment for DMD, according to a new study published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. [More]
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