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Current Muscular Dystrophy News and Events, Muscular Dystrophy News Articles.
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Researchers confirm prenatal heart defects in spinal muscular atrophy cases
University of Missouri researchers believe they have found a critical piece of the puzzle for the treatment of spinal muscular atrophy, or SMA -- the leading genetic cause of infantile death in the world. Nearly one in 6,000 births has SMA, and it is estimated that nearly one in 30 to 40 people have the trait that leads to SMA. (2010-09-28)

Study links normal function of protein, not its build up inside cells, to death of neurons
A study led by St. Jude Children's Research Hospital investigators links the muscle weakness and other symptoms of a rare neurodegenerative disease to a misstep in functioning of a normal protein, rather than its build-up inside cells. (2010-09-22)

Brown licenses potential muscular dystrophy treatment to Tivorsan Pharmaceuticals
Duchenne muscular dystrophy affects only young boys, who begin to show symptoms when they are of preschool age. The disease is invariably fatal. Justin Fallon has led a research team that has developed a novel therapy in mice. Brown University has licensed the technology to Tivorsan Pharamaceuticals, hoping to spur development of a treatment for humans. (2010-09-21)

Nature study shows how molecules escape from the nucleus
By constructing a microscope apparatus that achieves resolution never before possible in living cells, researchers at Albert Einstein College of Medicine of Yeshiva University have illuminated the molecular interactions that occur during one of the most important (2010-09-15)

Blacks with muscular dystrophy die 10-12 years younger than whites: New study
African-Americans with muscular dystrophy die 10-12 years younger than their white counterparts, according to a new research article in Neurology. The black-white mortality gap, calculated from 20 years of data, is among the largest ever observed in the annals of research into racial disparities in health care and is getting wider, says an accompanying editorial. The editorial says inequities in health delivery and in insurance coverage may explain the gap. (2010-09-13)

White Americans living longer with muscular dystrophy than African-Americans
A new study shows that white men and boys are living longer with muscular dystrophy due to technological advances in recent years, but that the lives of African-American men and boys with muscular dystrophy have not been extended at the same rate. The research will be published in the Sept. 14, 2010, issue of Neurology, the medical journal of the American Academy of Neurology. (2010-09-13)

Spinal muscular atrophy research team receives Pepsi Refresh funds from Sophia's Cure Foundation
Brian Kaspar, Ph.D., principal investigator in the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital, along with a team of Spinal Muscular Atrophy researchers and clinicians, recently received a $250,000 grant for SMA research and clinic development from Sophia's Cure Foundation via the Pepsi Refresh Project. (2010-09-08)

Rochester leads international effort to improve muscular dystrophy treatment
A large international study aimed at improving the care of muscular dystrophy patients worldwide is being launched by physicians, physical therapists, and researchers at the University of Rochester Medical Center, along with counterparts at 41 other institutions around the world. The study will compare treatments for boys with Duchenne muscular dystrophy, the most common form of the disease that affects children. (2010-09-03)

Induced pluripotent stem cell retain an inactivated X chromosome
Female induced pluripotent stem cells, reprogrammed from human skin cells into cells that have the embryonic-like potential to become any cell in the body, retain an inactive X chromosome, stem cell researchers at UCLA have found. (2010-09-02)

$7 million grant designates Nationwide Children's as Wellstone Center
A $7 million grant from the National Institutes of Health will help researchers at Nationwide Children's Hospital translate new scientific findings and technological developments into novel treatments for the muscular dystrophies. The grant designates Nationwide Children's Hospital as a Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, one of three national award recipients in 2010. (2010-08-26)

Gene involved in Fuchs corneal dystrophy is found
A 13-member research team led by University of Oregon scientist Dr. Albert O. Edwards has found a gene likely responsible for Fuchs corneal dystrophy, an inheritable genetic disorder and leading cause of corneal transplant operations. (2010-08-25)

Physiotherapy after surgery -- the best solution for shoulder problems
Most patients who receive physiotherapy after surgery experience that pain is reduced by a half within a few months. Most of them are free of pain after one to two years. This is the conclusion of a thesis presented at the University of Gothenburg, Sweden. (2010-08-23)

Discovery opens door to therapeutic development for FSH muscular dystrophy
Scientists are closer to understanding what triggers muscle damage in one of the most common forms of muscular dystrophy, called facioscapulohumeral muscular dystrophy. The new research presents a model of the disease that ties together many complex findings, and will allow researchers to test new theories and potential new treatments. (2010-08-19)

Scientists pinpoint earliest steps of common form of muscular dystrophy
Nearly two decades after they identified the specific genetic flaw that causes a common type of muscular dystrophy, scientists believe they have figured out how that flaw brings about the disease. The finding by an international team of researchers settles a longstanding question about the roots of facioscapulohumeral muscular dystrophy, or FSHD, and points to a new therapeutic target. (2010-08-19)

International research team closes in on cause of common form of muscular dystrophy
An international team of researchers that includes investigators from Fred Hutchinson Cancer Research Center has made a critical advance in determining the cause of a common form of muscular dystrophy known as facioscapulohumeral dystrophy, or FSHD. (2010-08-19)

Building muscle doesn't require lifting heavy weights: study
A new study shows that building muscle depends on achieving muscle fatigue not on pumping heavy weights as previously believed. (2010-08-11)

Parent Project Muscular Dystrophy awards $600K to Nationwide Children's for gene therapy study
Parent Project Muscular Dystrophy, the largest nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy, announced that PPMD will award a $600,000 grant to Nationwide Children's Hospital in Columbus, Ohio, to conduct clinical testing of a promising gene therapy technique for muscle disease. (2010-08-10)

Solving the mystery of bone loss from drug for epilepsy and bipolar disorder
Scientists are reporting a possible explanation for the bone loss that may occur following long-term use of a medicine widely used to treat epilepsy, bipolar disorder and other conditions. The drug, valproate, appears to reduce the formation of two key proteins important for bone strength, they said. Their study, which offers a solution to a long-standing mystery, appears in ACS' monthly Journal of Proteome Research. (2010-08-04)

Stanford scientists develop new way to grow adult stem cells in culture
Researchers at the Stanford University School of Medicine have developed a technique they believe will help scientists overcome a major hurdle to the use of adult stem cells for treating muscular dystrophy and other muscle-wasting disorders that accompany aging or disease: They've found that growing muscle stem cells on a specially developed synthetic matrix that mimics the elasticity of real muscle allows them to maintain their self-renewing properties. (2010-07-15)

Children's National Medical Center receives first NIH CTSA given to a children's hospital
Children's National Medical Center, in partnership with The George Washington University Medical Center, has received a prestigious Clinical and Translational Science Award from the National Center for Research Resources of the National Institutes of Health. This award, which totals $20 million over five years, is the first CTSA given directly to a children's hospital. (2010-07-14)

CSHL researchers demonstrate efficacy of antisense therapy for spinal muscular atrophy
Scientists at Cold Spring Harbor Laboratory and California-based Isis Pharmaceuticals have succeeded in reversing symptoms of type III SMA, a relatively mild form of spinal muscular atrophy, in mice by introducing chemically modified RNA pieces called ASOs into their spinal cords. The ASOs fix the molecular mistake underlying SMA by redirecting a cellular editing process called alternative splicing. (2010-07-11)

JCI online early table of contents: July 1, 2010
This release contains summaries, links to PDFs, and contact information for the following newsworthy papers to be published online, July 1, 2010, in the JCI: Identifying preterm infants at risk of life-threatening conditions; Childhood obesity: possible new insight from mice; A tale of tribbles 3: understanding its role in type 2 diabetes; A Silencing genes that protect from cancer: a role for the protein hNaa10p; TGF-beta a DAB(2) hand at tumor promotion; and others. (2010-07-01)

Cheap, simple, noninvasive blood test may replace invasive diagnostic techniques in early pregnancy
Researchers in the Netherlands believe they are on the verge of developing a simple, prenatal blood test that would be able to detect accurately chromosomal abnormalities in the developing fetus. At present, the only reliable way to do this is through amniocentesis or chorionic villus sampling, both of which are invasive and carry the risk of triggering a miscarriage. (2010-06-29)

Emerging questions about the mechanisms that control muscle
There are major shifts underway in understanding the physiological mechanisms that control muscle contraction, a field that has been the focus of intense research for centuries. The latest Perspectives in General Physiology series presents key novel findings, unresolved questions, and clinical implications of essential muscle processes that are controlled by distinct and complex multiprotein macromolecular machines. The Perspectives appear in the July issue of the Journal of General Physiology. (2010-06-28)

Team led by LA BioMed scientist develops novel approach to study neurological disorders
A team led by a LA BioMed researcher developed a novel approach to efficiently identify disease relevant genes in human complex neurological disorders and demonstrated the effectiveness of this method by applying it to amyotrophic lateral sclerosis, or Lou Gehrig's disease. (2010-06-24)

University of Utah researchers fight genetic killer of infants and toddlers
With the generous support of Families of Spinal Muscular Atrophy, researchers in the University of Utah Department of Neurology are making significant headway in the fight against the disease. Once very poorly understood, SMA is now considered one of the genetic conditions closest to finding an effective treatment. (2010-06-17)

Late-onset Pompe patients in US begin receiving new therapy
The first commercially available treatment in the United States for a rare form of muscular dystrophy called late-onset Pompe disease was administered at the University of Florida Health Science Center on Wednesday. People with Pompe disease lack an enzyme to process sugars and starches that are stored in the body as glycogen. The glycogen accumulates and destroys muscle cells, particularly those of the heart and respiratory muscles. (2010-06-16)

New therapy offers hope to spinal muscular atrophy patients
Children who suffer from the devastating disease spinal muscular atrophy are set to benefit from a new breakthrough in therapy developments by researchers at the University of Sheffield. (2010-06-09)

Jumping genes provide extensive 'raw material' for evolution, Penn study finds
Using high-throughput sequencing to map the locations of a common type of jumping gene within a person's entire genome, researchers found extensive variation in these locations among the individuals they studied, further underscoring the role of these errant genes in maintaining genetic diversity. The investigators determined that any two peoples' genomes differ at roughly 285 sites out of the 1,139 sites studied. (2010-06-01)

Researchers validate a new test for assessing children's and teenagers' fitness to prevent morbidity
Researchers from the University of Granada have concluded that either being overweight or being too thin can affect negatively teenagers' health. For the purpose of this study, 3,000 Spanish teenagers, 3,500 European teenagers and an additional sample of 126 teenagers from Granada were analyzed. (2010-05-27)

Tips from the American Journal of Pathology
The following highlights summarize research articles that are published in the June 2010 issue of the American Journal of Pathology. (2010-05-26)

Trans-Atlantic agreement a boost to neuroscience research
Neurological research and clinical care received a significant boost today as Imperial College London and McGill University of Montreal entered an agreement enabling them to work more closely together in this field. (2010-05-10)

NIH awards $7.5 million to study MRI as a tool to evaluate children with muscular dystrophy
Scientists from four institutions, including the University of Florida, Oregon Health and Science University, Children's Hospital of Philadelphia and the University of Pennsylvania, will assess whether MRI technology can be used as a precise, noninvasive measure of muscle tissue in children with Duchenne muscular dystrophy. Understanding how the disease affects muscle tissue could help facilitate the testing of new therapies in clinical trials. (2010-05-07)

Caught on tape: Muscle stem cells captured on video by MU researcher
Now, University of Missouri researchers have used time-lapse photography to document satellite cell movements and behaviors when they interact with their (2010-05-06)

Doctors use ultrasound to diagnose possible muscular trauma in professional athletes on-site
Doctors can use ultrasonography (ultrasound) to evaluate and diagnose muscular trauma in professional athletes on-site, which helps them to determine whether or not a player's injuries are severe enough to take them out of the game, according to a study to be presented at the ARRS 2010 Annual Meeting in San Diego, Calif. (2010-05-06)

Heart drug effective for treating symptom of muscular dystrophy
A medication most often used to treat heart arrhythmias also reduces a central symptom of myotonic dystrophy, the most common type of muscular dystrophy in adults. The findings about the medication mexiletine -- a chemical cousin of lidocaine -- were published May 4 in the journal Neurology. (2010-05-04)

MU researcher developing test for swallowing disorder treatments
One of the silent, and most serious, symptoms of Lou Gehrig's disease is losing the ability to swallow. Swallowing impairment, or dysphagia, affects about 500,000 people annually in the US, but little is known about the disorder. Now, a University of Missouri researcher is developing a test that might help pinpoint the neurological or physiological origins of swallowing disorders, leading to possible life-saving treatments. (2010-04-28)

Drug may treat cystic fibrosis, other diseases caused by 'nonsense mutations,' UAB researcher says
A renowned researcher on a select group of genetic alterations called nonsense mutations says scientists are now closer to producing drugs that will fix this disruption. UAB's David Bedwell, Ph.D., says, (2010-04-26)

New hope exists in treating inherited disease by suppressing DNA mutations
Genetic mutation can disrupt the way human cells make proteins, which in turn leads to inherited disease. According to one researcher, scientists are closer than ever to producing drugs that fix this disrupted-protein pathway and drastically improving treatment of genetic disease. (2010-04-26)

Researchers prove the gene responsible for Duchenne muscular dystrophy can be repaired
Researchers from Université Laval's Faculty of Medicine and the CHUQ Research Center have proven that it is possible to repair the defective gene responsible for Duchenne muscular dystrophy. The team, led by Professor Jacques P. Tremblay, is presenting its new therapeutic approach in an article published today in the online version of the scientific journal Gene Therapy. (2010-04-15)

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