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Research News

[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]
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 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]
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]
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]
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]
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
[title]
MDA Webinars Focus on ALS Medical Management, Research, Advocacy
July 01, 2010
MDA hosted three ALS-related webinars (Internet-based seminars) in May, in conjunction with National ALS Awareness Month. These webinars, which allow participants to direct questions to the featured
[title]
News from the ALS TDI
July 01, 2010
In continuing efforts to refine its preclinical drug development program, the ALS Therapy Development Institute (ALS TDI) of Cambridge, Mass., has added a new research mouse model to its operations,
[title]
ALS Research Roundup July-August 2010
July 01, 2010
Neuraltus targeting immune system cells Neuraltus Pharmaceuticals, of Palo Alto, Calif., is developing a small molecule whose target is regulation of immune system cells believed to contribute to
[title]
Brain-Computer Interface Reads Brainwaves
June 30, 2010
Note: This article was titled "The Thinking Cap" in the print edition of the July-August 2010 MDA/ALS Newsmagazine. Cathy Wolf of Katonah, N.Y., is able to manage only a small amount of muscle
[title]
Participants Sought for Three ALS Trials
June 23, 2010
Research scientists are seeking individuals interested in participating in any of the following three trials in ALS. High-fat, high-calorie diets Enrollment is open in an MDA-supported study
[title]
First US Exon-Skipping Trial Opens
June 18, 2010
Update (Nov. 5, 2012): Results announced in November 2012 showed the drug, now called drisapersen, reached blood levels approximately proportional to the injected dose at two of the three dosage
[title]
The Flip of a Switch
June 15, 2010
Neuraltus Pharmaceuticals of Palo Alto, Calif., is developing a small molecule whose target is regulation of immune system cells believed to contribute to neuroinflammation and disease progression in
[title]
ALS Ceftriaxone Trial Still Open
June 11, 2010
A 600-person trial of intravenous ceftriaxone, an antibiotic in the cephalosporin family that's approved to treat certain types of infections, is still looking for participants at 53 sites in the
[title]
DMD Trial: AVI4658 Increased Dystrophin Production
June 02, 2010
The biopharmaceutical company AVI BioPharma has announced additional encouraging results from its clinical trial of AVI4658, an experimental treatment for Duchenne muscular dystrophy (DMD). The new
[title]
SOD1 Versus Other ALS: Apples and Oranges?
May 19, 2010
Scientists at Northwestern University Feinberg School of Medicine in Chicago have announced new findings showing some forms of familial and nonfamilial ("sporadic") amyotrophic lateral sclerosis (ALS
[title]
MDA's ALS Clinical Research Network: Streamlining the Search for a Cure
May 01, 2010
The MDA ALS Clinical Research Network, a nationwide consortium of MDA/ALS centers, is up and running, helping streamline the search for treatments for ALS. The network provides an “unprecedented”
[title]
FALS Under the Microscope
May 01, 2010
Although familial, or inherited, ALS (FALS) and the sporadic (uninherited) form of the disease demonstrate similar progression patterns after symptoms appear, the causes for sporadic ALS remain

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