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Research News

[title]
ALS Research Briefs
September 13, 2010
DNA variants Variant sequences of DNA within a small region of chromosome 9 have been found to be associated with sporadic ALS (ALS without a family history) in a study that compared samples from
[title]
DMD Clinical Research Network Studying Dystrophin-Deficient Heart
September 09, 2010
Update (Jan. 23, 2013): As of early 2013, MDA's DMD Clinical Research Network includes these five sites: University of California, Davis (UC Davis); Nemours Children's Hospital in Orlando, Fla.;
[title]
Decoy Receptor Lures Myostatin, Helps Muscles
September 07, 2010
Luring away myostatin, and possibly other proteins that inhibit muscle growth, using a gene for a "decoy receptor" increased muscle size and strength in mice with a muscle disease resembling human
[title]
Leuprorelin Fails to Improve Swallowing in SBMA
August 30, 2010
 Reducing testosterone levels in a large-scale trial in men with spinal-bulbar muscular atrophy (SBMA, or Kennedy disease) did not significantly affect swallowing function, despite earlier
[title]
Nationwide Children's Hospital Named MD Research Center
August 27, 2010
The National Institutes of Health (NIH) has awarded $7 million to Nationwide Children's Hospital in Columbus, Ohio, and designated the institution a Paul D. Wellstone Muscular Dystrophy Cooperative
[title]
Study Seeks People With Uncertain MD Diagnoses
August 27, 2010
A study to determine the early features of late-onset Pompe disease (acid maltase deficiency) is seeking 250 adults who have a clinical diagnosis of unclassified limb-girdle muscular dystrophy (LGMD
[title]
Research Briefs: ALS, CMD, FA and SMA
August 27, 2010
Amyotrophic lateral sclerosis (ALS) Mutations in the gene for the FUS protein, recently identified as a cause of amyotrophic lateral sclerosis (ALS), have been found to cause the FUS protein to
[title]
ALS Research Briefs
August 27, 2010
Biogen Idec and Knopp Neurosciences announced Aug. 18, 2010, that they have entered into an agreement to continue developing KNS-760704 (dexpramipexole) as an experimental treatment for ALS. The drug
[title]
Expanded Ataxin 2 Genes a Major Contributor to ALS Risk
August 25, 2010
Scientists working in the United States and Germany have uncovered what appears to be the most common genetic contributor to amyotrophic lateral sclerosis so far identified. The genetic factor is a
[title]
Research Briefs: ALS, BMD, DMD, MMD, SMA, Muscle Regeneration
August 20, 2010
Amytrophic lateral sclerosis (ALS) Biogen Idec and Knopp Neurosciences announced Aug. 18, 2010, that they have entered into an agreement to continue developing KNS-760704 (dexpramipexole) as an
[title]
Not One But Two DNA Changes Are Needed to Cause FSHD
August 19, 2010
Facioscapulohumeral muscular dystrophy (FSHD) requires the presence of not one but two genetic changes, both on chromosome 4, before it causes its characteristic symptoms — weakness starting in the
[title]
ALS Experts Urge Caution Regarding Head Injury Findings
August 18, 2010
A new study has claimed professional athletes who have sustained repeated head injuries and developed what's known as "chronic traumatic encephalopathy" (CTE) may also be at higher-than-average risk
[title]
MDA Awards Nearly $3.5 Million in New ALS Grants
August 17, 2010
MDA has awarded 10 grants totaling nearly $3.5 million to fund research projects focused on uncovering the causes of, and developing therapies for, ALS. The new grants went to investigators at labs
[title]
MDA Awards More Than $14 Million in Research Grants
August 17, 2010
MDA has awarded 38 new research grants totaling more than $14 million and covering more than a dozen neuromuscular diseases.  MDA's Board of Directors met in Los Angeles July 16, where it reviewed
[title]
More FUS-Related ALS Cases Found
August 13, 2010
Mutations in the gene for a protein called FUS may be a more widely distributed cause of amyotrophic lateral sclerosis (ALS) than previously recognized, according to three separate reports, all
[title]
Research Briefs: ALS, DMD, MTM and SMA
August 12, 2010
Duchenne muscular dystrophy Acceleron Pharma announced Aug. 4 that it has received fast track designation from the U.S. Food and Drug Administration (FDA) for its experimental compound ACE031 for the
[title]
Blocking a Protein Extends Survival in ALS Mice
August 11, 2010
Elevated levels of an immune-system protein called interleukin-1-beta (IL-1-beta) exacerbates a disease in mice that closely resembles human amyotrophic lateral sclerosis (ALS), and blocking this
[title]
August is SMA Awareness Month
August 06, 2010
August is National Spinal Muscular Atrophy Awareness Month, and research in this disease has never been more promising. Major themes in spinal muscular atrophy (SMA) research include increasing
[title]
ALS-Causing TDP43 Overstays Its Welcome
August 04, 2010
At least some of the mutations in the TDP43 protein that are known to lead to ALS cause the TDP43 protein to be more stable than usual and change its interactions with other cellular proteins, say
[title]
ALS TDI Posts Quarterly Research Update
August 02, 2010
The ALS Therapy Development Institute’s second-quarter Research Update discussed new thinking about how ALS develops and progresses, and described progress in testing nearly 30 compounds targeting
[title]
Study to Probe Oxidative Stress in People With ALS
July 26, 2010
More than 400 people who recently received an ALS diagnosis are being sought for a large study of a cell-damaging phenomenon called "oxidative stress," common in ALS and other degenerative diseases.
[title]
Utah Researchers Seek CMD Families
July 22, 2010
Families affected by either of two forms of congenital muscular dystrophy (CMD), known as Ullrich CMD and Bethlem myopathy, are invited to help with an MDA-supported data collection project at the
[title]
Nonwalkers With SMA At High Risk for Weight Gain
July 22, 2010
Children and adults with types 2 and 3 spinal muscular atrophy (SMA) who are no longer walking have an increased risk of being overnourished and overweight if they otherwise have relatively good
[title]
Antisense Treatment Restores Full-Length SMN in SMA Mice
July 14, 2010
Scientists have found that mice with a disease resembling a mild form of spinal muscular atrophy (SMA) known as SMA type 3 showed more production of a needed protein in their spinal cords and more
[title]
Of Mice and Men: ALS TDI Discusses ALS Research Animals
July 06, 2010
Sean Scott, the late president of the ALS Therapy Development Institute (ALS TDI) in Cambridge, Mass., used to compare ALS to a building fire in which firemen show up focused on one thing: putting

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