Popular Muscular Dystrophy News and Current Events

Popular Muscular Dystrophy News and Current Events, Muscular Dystrophy News Articles.
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University of Minnesota research discovers inhibitor to reverse toxic DUX4 effects
New University of Minnesota Medical School research identifies an inhibitor that protects cells from toxic effects associated with facioscapulohumeral muscular dystrophy in cells and mice. (2019-09-11)

Improving academic performance with physical fitness
Physical fitness in childhood and adolescence is beneficial for both physical and mental health throughout life. However, a growing body of evidence suggests that it may also play a key role in brain health and academic performance. In a new study scheduled for publication in the Journal of Pediatrics, researchers studied the independent and combined influence of components of physical fitness on academic performance. (2014-06-19)

Hi-res view of protein complex shows how it breaks up protein tangles
A new, high-resolution view of the structure of Hsp104 (heat shock protein 104), a natural yeast protein nanomachine with six subunits, may show news ways to dismantle harmful protein clumps in disease. (2017-06-15)

Ball games and circuit strength training boost bone health in schoolchildren
The type of exercise that children get in school does make a difference. This is shown by a major Danish study from researchers at the University of Southern Denmark and University of Copenhagen. Eight to ten-year-old schoolchildren develop stronger bones, increased muscular strength and improved balance when ball games or circuit training are on the timetable. (2018-02-08)

CRISPR helps find new genetic suspects behind ALS/FTD
NIH-funded researchers used the gene editing tool CRISPR to rapidly identify genes in the human genome that might modify the severity of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) caused by mutations in a gene called C9orf72. The results of the search uncovered a new set of genes that may hasten neuron death during the disease. (2018-03-12)

Therapy for muscular dystrophy-caused heart failure also improves muscle function in mice
Injections of cardiac progenitor cells help reverse the fatal heart disease caused by Duchenne muscular dystrophy and also lead to improved limb strength and movement ability, a new study shows. The study, published today in Stem Cell Reports, showed that when researchers injected cardiosphere-derived cells (CDCs) into the hearts of laboratory mice with muscular dystrophy, heart function improved along with a marked increase in exercise capacity. (2018-02-22)

New drug improves motor function of children with genetic disorder
Children with later-onset spinal muscular atrophy (SMA) were more likely to show gains in motor function when treated with a new medication compared to children receiving a sham procedure, according to a study published today in the New England Journal of Medicine. The study demonstrates the impact the drug, nusinersen, can have on older patients with this progressive neuromuscular disorder. (2018-02-14)

Study finds genetic mutation causes 'vicious cycle' in most common form of ALS
University of Michigan-led research brings scientists one step closer to understanding the development of neurodegenerative disorders such as ALS. A study published today in Nature Communications details what the researchers describe as a vicious cycle of toxic protein production set in motion by cell stress. (2017-12-08)

University of Minnesota researchers replicate FSH muscular dystrophy in mice
A new study published in the journal Nature Communications describes a breakthrough in research related to facioscapulohumeral muscular dystrophy (FSHD). The debilitating genetic disease -- which has no approved treatment -- affects an estimated 38,000 Americans and causes muscle degeneration. Scientists inserted into mice a gene called DUX4, which is believed to cause FSHD in humans. When they activated the gene in mice skeletal muscle cells, the animals developed a muscular dystrophy with key features of FSHD. (2017-09-15)

Breakthrough brings gene-editing medicine one step closer to patient applications
Researchers at the University of Alberta have discovered a way to greatly improve the accuracy of gene-editing technology by replacing the natural guide molecule it uses with a synthetic one called a bridged nucleic acid, or BNA. The research promises to bring the technology much closer to therapeutic reality. (2018-04-13)

Research suggests alternative treatment for beta blocker intolerant heart attack patients
Beta blockers have become a prescription drug staple for recovering heart attack patients. However, these blood pressure-reducing medications cannot be tolerated by many patients who are at higher risk for developing cardiovascular disease, including those with chronic obstructive pulmonary disease (COPD) and asthma, the elderly, and diabetics. As seen in the March 26 issue of Thyroid, researchers at New York Institute of Technology College of Osteopathic Medicine (NYITCOM) now pose a new treatment for patients with beta blocker intolerance: thyroid hormone therapy. (2018-04-10)

Preliminary study suggests drug may help babies with spinal muscular atrophy
A preliminary study suggests that an investigational drug may help increase protein levels in babies with spinal muscular atrophy. The open-label study is released today and will be presented at the American Academy of Neurology's 70th Annual Meeting in Los Angeles, April 21-27, 2018. (2018-04-18)

New advances in medication for muscle disease in children
Spinraza, the gene therapy medication, also provides significant improvements in cases with the next most severe form of neuromuscular disease, spinal muscular atrophy (SMA), which afflicts children from 6 to 18 months of age. That is shown by a study published in the New England Journal of Medicine (NEJM). (2018-02-26)

From black hat to white hat: Findings tip assumptions about TAK1 in muscle growth
Convention was that the signaling protein, transforming growth factor-ß-activated kinase 1 (TAK1) is detrimental to muscle health since it activates pathways associated with muscle wasting. However deactivating TAK1 did not preserve muscle health as expected, but resulted in the opposite effect: muscle wasting. (2018-02-08)

UCLA researchers create skeletal muscle from stem cells
UCLA scientists have developed a new strategy to efficiently isolate, mature and transplant skeletal muscle cells created from human pluripotent stem cells, which can produce all cell types of the body. The findings are a major step towards developing a stem cell replacement therapy for muscle diseases including Duchenne Muscular Dystrophy, which affects approximately 1 in 5,000 boys in the US and is the most common fatal childhood genetic disease. (2017-12-18)

Salk scientists find power switch for muscles
ERRγ gene enables endurance exercise and repairs type of damage seen in neuromuscular diseases. (2018-03-06)

Cell therapy improves heart function, upper limb strength in duchenne muscular dystrophy
After boys and young men with Duchenne muscular dystrophy received cardiac progenitor cell infusions, medical tests indicated that the patients' hearts appeared improved, results from a new study show. Patients in the study also scored higher on arm strength tests after receiving the cell infusions. (2017-11-15)

Scientists discover a key function of als-linked protein
The protein FUS, whose mutation or disruption causes many cases of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), works as a central component of one of the most important regulatory systems in cells, according to a new study in Molecular Cell from scientists at Johns Hopkins Bloomberg School of Public Health. (2018-03-08)

University of Minnesota research derives muscle stem cells from teratomas
Researchers at the University of Minnesota Medical School have developed a process to regenerate skeletal muscle cells in mice with muscular dystrophy. (2018-07-05)

Gene therapy cassettes improved for muscular dystrophy
Experimental gene therapy cassettes for Duchenne muscular dystrophy have been modified to deliver better performance. The cassettes, which carry the therapy into muscle cells, contain newer versions of a miniaturized treatment gene. Earlier versions of the treatment cassettes did significantly enhance muscle function in previous lab studies, but did so incompletely. That's partly because the huge dystrophin gene has to be condensed to fit inside the transport virus. (2019-02-01)

Analysis of global duchenne muscular dystrophy patients registry underscores
Duchenne muscular dystrophy (DMD) is a a rare and fatal genetically-inherited degenerative neuromuscular disease that affects one in 5,000 newborn boys. (2018-02-01)

What happens in the bodies of ALS patients?
Lara Marrone and Jared Sterneckert from the Centre for Regenerative Therapies Dresden (CRTD) at Technische Universität Dresden (TUD), together with collaborating scientists from Germany, Italy, the Netherlands, and the USA, have now discovered that interactions between RNA-binding proteins are more critical to ALS pathogenesis than previously thought. (2019-04-15)

CRISPR-Cas9 technique targeting epigenetics reverses disease in mice
In a paper appearing in the journal Cell on Dec. 7, scientists at the Salk Institute report a modified CRISPR-Cas9 technique that alters the activity, rather than the underlying sequence, of disease-associated genes. The researchers demonstrate that this technique can be used in mice to treat several different diseases. (2017-12-07)

Researchers illustrate how muscle growth inhibitor is activated, could aid in treating ALS
Researchers at the University of Cincinnati (UC) College of Medicine are part of an international team that has identified how the inactive or latent form of GDF8, a signaling protein also known as myostatin responsible for limiting muscle, is activated. (2018-01-19)

Children's Hospital scientists achieve repair of injured heart muscle in lab tests of stem cells
Researchers at Children's Hospital of Pittsburgh of UPMC have been able to effectively repair damaged heart muscle in an animal model using a novel population of stem cells they discovered that is derived from human skeletal muscle tissue. (2008-11-25)

Researchers track muscle stem cell dynamics in response to injury and aging
A new study led by SBP describes the biology behind why muscle stem cells respond differently to aging or injury. The findings, published in Cell Stem Cell, have important implications for the normal wear and tear of aging. (2017-12-14)

Engineers grow functioning human muscle from skin cells
Duke engineers have grown the first functioning human muscle from non-muscle cells -- skin cells reverted to their primordial stem cell state. The ability to start from cellular scratch using non-muscle tissue will allow scientists to grow far more muscle cells, provide an easier path to genome editing and cellular therapies, and develop individually tailored models of rare muscle diseases for drug discovery and basic biology studies. (2018-01-09)

Unusual protein modification involved in muscular dystrophy, cancer
With the discovery of a new type of chemical modification on an important muscle protein, a University of Iowa study improves understanding of certain muscular dystrophies and could potentially lead to new treatments for the conditions. (2009-12-31)

Targeting astrocytes slows disease progression in ALS
In what the researchers say could be promising news in the quest to find a therapy to slow the progression of amyotrophic lateral sclerosis, or Lou Gehrig's disease, scientists at the University of California, San Diego School of Medicine have shown that targeting neuronal support cells called astrocytes sharply slows disease progression in mice. (2008-02-03)

Supply bottleneck impairs nerve function
Impaired transport processes in neurons contribute to diseases such as amyotrophic lateral sclerosis (AML). Würzburg scientists have now identified key actors in these processes. (2018-03-08)

Huntington's Disease Mouse Model To Be Distributed By Jackson Laboratory
The first strain of mouse genetically engineered to model major symptoms of Huntington's disease -- an inherited, degenerative brain disease that affects an estimated 30,000 Americans -- is now undergoing processing at The Jackson Laboratory for distribution to researchers worldwide (1997-04-02)

Designer molecule points to treatment for diseases caused by DNA repeats
Using a molecule designed to overcome a roadblock formed by a common type of genetic flaw, researchers at the University of Wisconsin-Madison have made progress towards novel molecular treatments for Friedreich's ataxia -- a rare but fatal disorder -- in the laboratory dish and in animals. (2017-11-30)

Chronic inflammation plays critical role in sustained delivery of new MD therapy
Macrophages, a type of white blood cell involved in inflammation, readily take up a newly approved medication for Duchenne muscular dystrophy and promote its sustained delivery to regenerating muscle fibers long after the drug has disappeared from circulation, an experimental model study finds. (2017-10-16)

Novel therapy delays muscle atrophy in Lou Gehrig's disease model
Supplementing a single protein found in the spinal cord could help prevent symptoms of Lou Gehrig's disease, according to a new study out of Case Western Reserve University School of Medicine. Researchers found high levels of the protein -- called mitofusion 2 or Mfn2 -- prevented nerve degeneration, muscle atrophy, and paralysis in a mouse model of the disease. Since Mfn2 is often depleted during Lou Gehrig's, the new study suggests supplementing it could be a novel therapeutic approach for the disease. (2018-07-12)

Nanostructures created at UCLA could make gene therapies safer, faster & more affordable
UCLA scientists have developed a new method that utilizes microscopic splinter-like structures called 'nanospears' for the targeted delivery of biomolecules such as genes straight to patient cells. (2018-03-14)

Tamoxifen and raloxifene slow down the progression of muscular dystrophy
Steroids are currently the only available treatment to reduce the repetitive cycles of inflammation and disease progression associated with functional deterioration in patients with muscular dystrophy (MD). A study reported in The American Journal of Pathology showed that a new treatment approach using the selective estrogen receptor modulators (SERMs) tamoxifen and raloxifene significantly improved cardiac, respiratory, and skeletal muscle functions and increased bone density in both male and female mice with the same gene defects as a subset of patients with MD. (2018-03-20)

Researchers overcome obstacle for future stem cell therapies
Researchers have discovered a new technique that overcomes one of the major challenges of stem cell therapy. A drug, co-created by UBC researchers, might overcome one of the major challenges of stem cell therapy -- their tendency to differentiate, becoming specific tissue cells too early and too quickly. (2018-01-25)

Device that measures cell strength could help identify drugs for asthma, hypertension
Engineers, doctors and scientists at UCLA and Rutgers University have developed a tool that measures the physical strength of individual cells 100 times faster than current technologies. (2018-02-09)

Not all Europeans receive the same care for Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD), a progressive muscle disease affecting one in 3,800-6,300 live male births and leads to ambulatory loss, respiratory problems, cardiomyopathy, and early death of patients in their 20s or 30s. While incurable, multidisciplinary treatment can raise life expectancy into the fourth decade. However, in a survey across seven European countries, researchers found striking differences in access to appropriate care. There were significant inequities between different countries and different age groups, which would likely lead to different health outcomes. (2017-01-03)

CU researchers identify potential treatment for diastolic dysfunction in heart failure
Researchers at the University of Colorado School of Medicine have identified a potential treatment target for patients with a common type of heart failure. In a study published in the Feb. 7 issue of the journal Science Translational Medicine, the researchers tested the effect of an investigational drug called givinostat in treating diastolic dysfunction, which is a heart relaxation abnormality that contributes to heart failure with preserved ejection fraction (HFpEF). (2018-02-07)

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