Quest Magazine

Phase 3 Trial of Dexpramipexole Launched in ALS

Update 8/31/11: Enrollment screening for this study has closed to new prospective participants.

Research Briefs: DMD Exon Skipping

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 the United States and elsewhere.

Exon skipping for DMD is a strategy that coaxes muscle fibers to ignore, or "skip," the genetic instructions for certain parts of the dystrophin gene so that functional dystrophin protein can be made despite the presence of a genetic mutation.

MDA Awards Grant to Glialogix to Test Potential ALS Treatment

MDA is funding research into a potential treatment for amyotrophic lateral sclerosis (ALS) that inhibits excessive glutamate signaling in the brain and spinal cord.

DMD/BMD Drug Screen in Zebrafish Finds Three Candidates

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 Medical School, and Alaska Fisheries Science Center in Juneau.

The three chemicals, which already are approved for other medical uses, were found to increase survival and normalize muscle structure and function in the dystrophin-deficient fish without increasing  production of the muscle protein dystrophin. 

ALS Research Briefs

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 TDP43 are a known cause of ALS, and TDP43 is depleted in the nucleus and enriched in the cytoplasm (main cell compartment) in ALS-affected nerve cells, even when no TDP43 mutations exist. Understanding TDP43's many functions is likely to improve understanding of ALS.

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FCC Surveying People with Disabilities about Calling 911

The Federal Communications Commission (FCC) is conducting an online national survey of individuals with disabilities to determine the most effective and efficient methods to access emergency services.

According to the FCC, a new emergency-access system is being developed that will allow people to reach 911 not only by using voice telephones, but text, video and other devices, some of which use the Internet.

MDA Awards Grant to Test Heat Shock Protein in ALS Mice

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 form to meet study standards.  The ability to reliably produce the compound in sufficient quantity and quality for testing purposes was one of a number of milestones that had to be met in order for the project to receive full funding.

MDA Conference Brings Together Researchers, Clinicians, Industry

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 Vegas.

Some 300 people attended the conference, the first in a planned series of such MDA-sponsored meetings that will emphasize new research and current medical care. The majority of presenters and many of the audience members were current or former MDA research grantees or physicians at MDA-supported clinics.

'Antisense' Ameliorates SMA Symptoms in Mice

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 protein throughout their spinal cords; developed bigger, stronger muscles; and survived longer than expected. 

IL10 Protein May Influence DMD Severity

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 reduce harmful inflammation and promote muscle regeneration in people with Duchenne muscular dystrophy (DMD), and potentially those with other forms of muscular dystrophy.

MDA supported James Tidball, director of the Duchenne Muscular Dystrophy Research Center at UCLA, for this work.

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