Quest Magazine

New Grant For LGMD2D Gene Therapy

Development of delivery of a therapeutic gene via the bloodstream to the thigh muscles in people with type 2D limb-girdle muscular dystrophy (LGMD2D) is proceeding on schedule, thanks in part to a new $458,814 grant from MDA to neurologist Jerry Mendell at Nationwide Children's Hospital in Columbus, Ohio.

MDA's Board of Directors approved the new funding, via the Association's translational research/MDA Venture Philanthropy program, on Dec. 4, 2009.

Exon-Skipping Drug Delivers Again

Interim results from a human clinical trial of the exon-skipping compound AVI4658 in boys with Duchenne muscular dystrophy (DMD) show that when the compound is delivered to the whole body via the bloodstream — rather than simply injected into a foot muscle as in a previous trial — it appears safe and leads to production of the missing muscle protein dystrophin.

New MDA Grant Will Help Develop FA Drug

Development of a promising experimental medication to treat Friedreich's ataxia (FA) is proceeding, with help from a $731,534 grant MDA awarded to Repligen Corp. of Waltham, Mass., this month (December 2009).

This is the second research grant that MDA has awarded to the small biopharmaceutical company, through the Association's translational (laboratory-to-clinic) research program.

Big Horses, Big Time

"Pa, I’m gettin' darn tired of goin’ down there and comin’ back with second place."

Robert Powell, 14, ordinarily is a young man of very few words. But when the subject is horse-pulling contests, it garners the full attention of this lad from Pleasuresville, Ky.

Internship Opportunities for College Students

Summer internships are available for college students with disabilities - but students must act quickly to apply.

Dec. 31, 2009 is the deadline to apply to the 2010 Emerging Leaders Summer Internship Program for College Students with Disabilities, a highly competitive program that places undergraduate and graduate students with disabilities in fulfilling summer internships and provides leadership development opportunities with many of America’s leading corporations.

ALS Research: Restoring Disrupted Connections

A molecule called microRNA 206, produced by muscle fibers after an injury to nerve cells, helps rebuild crucial nerve-muscle communications, say scientists at the University of Texas Southwestern Medical Center in Dallas and Harvard University. They say raising levels of microRNA 206 or amplifying its effects in some other way could become a new therapeutic avenue in amyotrophic lateral sclerosis (ALS).

They found that mice with an ALS-like disease fared worse without microRNA 206 than with it.

ALS Research: Restoring Disrupted Connections

A molecule called microRNA 206, produced by muscle fibers after an injury to nerve cells, helps rebuild crucial nerve-muscle communications, say scientists at the University of Texas Southwestern Medical Center in Dallas and Harvard University. They say raising levels of microRNA 206 or amplifying its effects in some other way could become a new therapeutic avenue in amyotrophic lateral sclerosis (ALS).

They found that mice with an ALS-like disease fared worse without microRNA 206 than with it.

Two ALS Drugs Show Early Promise

Two experimental compounds under development by different drug companies have shown encouraging early results,  according to reports given at the 20th International Symposium on ALS/MND in Berlin.

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International ALS Conference

ALS researchers from around the world gathered in Berlin, Germany, Dec. 8-10, to report on and discuss the latest research advances in this disease.

Daily updates from the symposium are available online at the Web sites of the Motor Neurone Disease Association (MNDA Symposium reports) and ALS Therapy Development Institute (ALS TDI Symposium reports). 

ALS Research: Poison Dirt?

New findings suggest a possible link between dust-dwelling bacterial toxins and an elevated incidence of ALS (amyotrophic lateral sclerosis) in Gulf War veterans.

The study blames cyanobacteria, microorganisms that live in desert sands and which can be inhaled when they’re kicked up in dust, such as when a convoy of military vehicles rumbles by. Cyanobacteria are common throughout the world in salt water, fresh water and soil.

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