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Research News

[title]
MDA Surveying Families about ‘Cost of Illness’
April 13, 2011
Most people affected by neuromuscular disease know all too well that their condition is costly. MDA wants to find out just how costly — and then translate those figures into terms that will speak
[title]
Online Games Help Fight Disease
March 31, 2011
Note: This article was updated Sept. 19, 2011. Video games have a reputation for being big time-wasters. But what if you could help solve scientific mysteries — such as unraveling the origins of a
[title]
Care Guidelines Issued for Congenital Muscular Dystrophies
March 31, 2011
Update (January 25, 2012): This story was updated to reflect the online availability of a CMD Family Guide. A panel of 82 international experts — including several MDA grantees and clinic directors
[title]
Research Briefs: CMT, IBM, LGMD, MTM/CNM, Pompe disease
March 31, 2011
Charcot-Marie-Tooth disease A two-year, large-scale trial of ascorbic acid (vitamin C) in people with type 1A Charcot-Marie-Tooth disease (CMT1A) conducted in Italy and the United Kingdom has found
[title]
Placebos and the Placebo Effect
March 31, 2011
A placebo is a sham, or fake, “medication” or “treatment” that, although inert, is designed to appear identical to an active actual or experimental treatment. Placebos can take the form of a “dummy”
[title]
Phase 3 Trial of Dexpramipexole Launched in ALS
March 30, 2011
Update 8/31/11: Enrollment screening for this study has closed to new prospective participants. "EMPOWER," a large-scale, global phase 3 clinical trial of a molecule called dexpramipexole in ALS (
[title]
Research Briefs: DMD Exon Skipping
March 29, 2011
sClinical trials that use compounds called antisense oligonucleotides to cause skipping of exon 51 of the dystrophin gene in individuals with Duchenne muscular dystrophy (DMD) are moving forward in
[title]
MDA Awards Grant to Glialogix to Test Potential ALS Treatment
March 24, 2011
MDA is funding research into a potential treatment for amyotrophic lateral sclerosis (ALS) that inhibits excessive glutamate signaling in the brain and spinal cord. The grant of $268,000 to Larkspur
[title]
DMD/BMD Drug Screen in Zebrafish Finds Three Candidates
March 24, 2011
Three small molecules that normalize muscle structure and function and improve survival in dystrophin-deficient zebrafish have been identified by scientists at Children's Hospital Boston, Harvard
[title]
ALS Research Briefs
March 23, 2011
TDP43 has unexpectedly extensive role Scientists have found that the protein TDP43 normally influences the synthesis of more than 1,500 other proteins, including itself. Mutations in the gene for
[title]
MDA Awards Grant to Test Heat Shock Protein in ALS Mice
March 21, 2011
UPDATE (April 9, 2012): MDA and ALS Biopharma agreed to terminate this project in April 2012, when ALS Biopharma determined that it was unable to manufacture its HSP70-based compound in a pure enough
[title]
MDA Conference Brings Together Researchers, Clinicians, Industry
March 18, 2011
Moving therapeutic strategies from the laboratory to clinical trials and ultimately to the market as treatments was the theme of the MDA National Scientific Conference held March 13-16, 2011, in Las
[title]
'Antisense' Ameliorates SMA Symptoms in Mice
March 15, 2011
A team of research scientists has found that mice with a disease resembling a severe form of spinal muscular atrophy (SMA) that were treated with a gene-modifying molecule produced more of a needed
[title]
MDA Awards Grant to Test Potential MG Treatment
March 14, 2011
MDA’s translational research program has announced it is funding research into a potential treatment for the autoimmune disorder myasthenia gravis (MG). The grant of $530,480 to University of
[title]
IL10 Protein May Influence DMD Severity
March 09, 2011
A team of researchers at the David Geffen School of Medicine at the University of California-Los Angeles (UCLA) has demonstrated that the naturally occurring protein interleukin 10 (IL10) may help
[title]
Reactivated Virus May Contribute to ALS
March 07, 2011
New research suggests that the DNA of a normally dormant virus may be playing a role in causing amyotrophic lateral sclerosis (ALS). If the findings are confirmed, they could ultimately have
[title]
ALS Research Briefs
March 03, 2011
Loss of TDP43's normal function could be ALS contributor New mouse studies suggest that loss of the normal function of the TDP43 protein, and not just an abnormal "toxic gain of function" of this
[title]
MDA Awards $2 Million to ALS TDI
March 02, 2011
MDA has awarded $2 million to the ALS Therapy Development Institute (ALS TDI) in Cambridge, Mass., to help scientists complete the preclinical testing of four promising new compounds in ALS TDI's
[title]
New Insights Shared at International ALS Meeting
March 01, 2011
New insights and perspectives on the biology of ALS, best practices in the care of individuals with the disease and future directions for development of ALS therapies were the central themes of the
[title]
ALS Research Roundup Spring 2011
March 01, 2011
ALS TDI expansion among 2011 goals Building on progress made in 2010, the MDA-supported ALS Therapy Development Institute (ALS TDI) has outlined a number of milestones it plans to meet in 2011.  
[title]
MDA Funds Eight New ALS Research Projects
March 01, 2011
MDA has awarded eight new grants totaling nearly $2.5 million to fund research into the causes of, and potential therapies for, ALS. The grants were reviewed and approved by the MDA Board of
[title]
Research Briefs: CMS, DMD, LGMD, Pompe, Stem Cells
February 25, 2011
Congenital myasthenic syndromes A multinational team of scientists has identified mutations in the gene for glutamine-fructose-6-phosphate transaminase 1 (GFPT1) as responsible for some forms of a
[title]
Research Briefs: CMT, CMS, DMD/BMD, FA, Pompe disease, SBMA
February 10, 2011
Charcot-Marie-Tooth disease Researchers supported in part by MDA have developed flow charts to guide genetic testing for the various subtypes of Charcot-Marie-Tooth disease (CMT), streamlining the
[title]
ALS Therapy Development Institute: Looking Ahead to 2011
February 10, 2011
New space for research, new mouse models of ALS and new technology to screen therapeutic agents all will play a role in 2011 at the ALS Therapy Development Institute (ALS TDI), said Steve Perrin, ALS
[title]
UCLA Researcher Receives MDA Grant to Develop DMD Drug
February 09, 2011
A new MDA translational research grant for $476,465 over three years will allow Carmen Bertoni at the University of California, Los Angeles (UCLA) to develop RTC13, an experimental compound designed

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