Quest Magazine

ALS Research: MDA Hosts 'Think Tank'

 

An all-day "think tank" to consider priorities for amyotrophic lateral sclerosis (ALS) research based on the latest information was conducted with ten of the leading scientists and clinicians in the field and senior MDA staff in Tucson, Ariz., on Saturday, Oct. 24, 2009.

Each specialist introduced his or her area of expertise and led an approximately 20-minute discussion, gathering input and answering questions from the other participants.

Vote Abbey for Gap Kid!

Abbey Umali needs your vote.

The vivacious 10-year-old, who is MDA’s National Goodwill Ambassador, is competing for the chance to add another title to her resume: Gap Kid.

Popular international retailer Gap has launched a nationwide search for the next faces for GapKids, its kids’ clothing line. Abbey, who has a form of Charcot-Marie-Tooth disease, is in the running and could use your help to win "fan favorite."

ALS Research: APC & ALS

A compound known as "activated protein C" (APC) that is already in use to treat severe bloodstream infections also may have benefit in ALS, according to a recently published report.

ALS Research: APC & ALS

A compound known as "activated protein C" (APC) that is already in use to treat severe bloodstream infections also may have benefit in ALS, according to a recently published report.

MDA Hosts One-Day ALS 'Think Tank'

Neurologist Valerie Cwik, senior executive vice president-research and medical director at MDA, and neurologist Stan Appel

DMD Research: US Exon Skipping Trial

The first human trial in the United States of a treatment strategy known as "exon skipping" for Duchenne muscular dystrophy (DMD) is scheduled to begin in March 2010 at Nationwide Children's Hospital in Columbus, Ohio, one of five elite centers comprising MDA's DMD Clinical Research Network.

First Human Exon Skipping Trial in US Planned for 2010

Drug Used to Treat Sepsis Shows Promise in Mice with ALS

A compound known as "activated protein C" (APC) that is already in use to treat severe bloodstream infections also may have benefit in ALS, according to a recently published report.

DMD Research: 'Remarkable' Results

Mice with a severe disease closely resembling human Duchenne muscular dystrophy (DMD) have responded extremely well to a new "exon skipping" compound that targets the specific dystrophin gene error these mice have.

Researchers noted "a remarkable prevention of the dystrophic pathology and improvement of the muscle function" in these severely affected mice, which lack both the dystrophin and utrophin muscle proteins. (Mice in previous exon skipping trials lacked only dystrophin, the protein missing in DMD.)

A Promising New Development for Treatment of Duchenne Muscular Dystrophy

A technique called exon skipping shows great potential to increase muscle strength and prolong life in people with a severe form of Duchenne muscular dystrophy (DMD). According to a study that included MDA-supported Stephen Wilton at the University of Western Australia, exon skipping improves production of a crucial muscle protein that’s missing in people with DMD. For the first time, these results were observed in mice with an especially severe form of muscular dystrophy.

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