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.

SLU scientists discover nuclear receptor that appears to play key role in muscle regeneration

Saint Louis University researchers report in Molecular Metabolism new findings that the nuclear receptor REV-ERB appears to play a key role in muscle regeneration, suggesting the receptor may be a good target for new drugs to treat a variety of muscle disorders and injuries.

14 Jun 2017

New approach could potentially prevent muscle wasting in muscular dystrophy and cancer

Monash University's Biomedicine Discovery Institute researchers have collaboratively developed a therapeutic approach that dramatically promotes the growth of muscle mass, which could potentially prevent muscle wasting in diseases including muscular dystrophy and cancer.

14 Jun 2017

Decades-old drug used to treat malaria reduces toxic protein linked to ALS

A new study finds that a decades-old drug used to treat malaria lowers levels of a biomarker linked to the inherited form of amyotrophic lateral sclerosis, also known as ALS and Lou Gehrig's Disease.

12 Jun 2017

Researchers find long-lasting benefits of gene therapy in dog model of myotubular myopathy

Researchers who previously showed that a gene therapy treatment could save the lives of dogs with a deadly disease called myotubular myopathy—a type of muscular dystrophy that affects the skeletal muscles—have found that the therapy is long-lasting.

7 Jun 2017

Researchers discover connection between processes that keep the heart from repairing itself

Heart muscle is one of the least renewable tissues in the body, which is one of the reasons that heart disease is the leading cause of death for both men and women in the United States, according to the Centers for Disease Control and Prevention.

5 Jun 2017

Scientists identify new gene that prompts muscle stem cells to fuse together

A detour on the road to regenerative medicine for people with muscular disorders is figuring out how to coax muscle stem cells to fuse together and form functioning skeletal muscle tissues.

1 Jun 2017

Weekly doses of steroids help hasten recovery in muscle injuries

Weekly doses of glucocorticoid steroids, such as prednisone, help speed recovery in muscle injuries, reports a new Northwestern Medicine study. The weekly steroids also repaired muscles damaged by muscular dystrophy.

17 May 2017

Preclinical data reinforces potential efficacy, durability of investigational gene therapy for DMD

Solid Biosciences announced today that new data from two preclinical studies reinforce the potential of its investigational microdystrophin gene therapy, SGT-001, to be an effective treatment approach for Duchenne muscular dystrophy.

11 May 2017

EPFL researchers discover protein that kick-starts gene expression in nascent embryos

The formation of a human embryo starts with the fertilization of the oocyte by the sperm cell. This yields the zygote, the primordial cell that carries one copy each of the maternal and paternal genomes.

2 May 2017

Actress Kiruna Stamell argues about gene editing with Dr Christopher Gyngell

Two papers published today by the Journal of the Royal Society of Medicine, debate gene editing and the health of future generations.

29 Apr 2017

NIH scientists discover mitochondrial 'circuit breaker' that prevents damage to the heart muscle

A team of scientists from the National Institutes of Health has discovered biological mechanisms that appear to prevent damage to the heart muscle's "power grid," the network of mitochondrial circuits that provide energy to cells.

18 Apr 2017

Researchers correct Duchenne muscular dystrophy using gene-editing alternative

Using the new gene-editing enzyme CRISPR-Cpf1, researchers at UT Southwestern Medical Center have successfully corrected Duchenne muscular dystrophy in human cells and mice in the lab.

13 Apr 2017

BMJ Innovations releases first collaborative definition of patient centricity co-created by AstraZeneca

AstraZeneca today announced that BMJ Innovations has published the first collaborative definition of patient centricity for the pharmaceutical industry.

10 Apr 2017

Researchers discover small protein essential for formation of skeletal muscle

UT Southwestern Medical Center researchers discovered a small protein named Myomixer essential for the formation of skeletal muscle – findings that could eventually help treat genetic diseases such as muscular dystrophy and other myopathies.

7 Apr 2017

Researchers achieve new step towards treatment of myotubular myopathy using gene therapy

A team of researchers in France, led by Dr. Ana Buj-Bello (Genethon/Inserm) and teams at the University of Washington and Harvard Medical School in the United States, achieved a new step towards the treatment of myotubular myopathy by gene therapy.

5 Apr 2017

Scientists develop novel technique to screen non-coding DNA of genome for diseases

Researchers have developed a method to swiftly screen the non-coding DNA of the human genome for links to diseases that are driven by changes in gene regulation.

4 Apr 2017

New research reveals surprising culprit involved in self-destruction of axons

In many neurodegenerative conditions — Parkinson's disease, amyotrophic lateral sclerosis (ALS) and peripheral neuropathy among them — an early defect is the loss of axons, the wiring of the nervous system.

24 Mar 2017

SLU researchers aim to tackle diabetes and obesity by capturing benefits of exercise in pill form

With a series of new grants, Saint Louis University researchers will tackle the twin epidemics of diabetes and obesity by tapping into the potential of two nuclear receptors that control muscle metabolism.

24 Mar 2017

Early treatment with heart failure drug can improve cardiac function in young boys with DMD

Researchers at The Ohio State University Ross Heart Hospital and Nationwide Children's Hospital have shown early treatment with the heart failure medication eplerenone can improve heart function in young boys with Duchenne muscular dystrophy (DMD) and stabilize heart function in older boys with the disease.

7 Mar 2017

New test could accelerate development of better treatments for Motor Neuron Disease

A cutting-edge test developed in South Australia has the potential to expedite the worldwide quest to develop better treatments, or even a cure, for the Motor Neuron Disease (MND).

24 Feb 2017