Duchenne Muscular Dystrophy (DMD)

Follistatin Genes Strengthen Muscles in Monkeys

Four macaque monkeys that received injections of genes for a protein called follistatin into upper leg muscles experienced pronounced and durable increases in muscle size and strength with no adverse effects, say researchers at Nationwide Children's Hospital in Columbus, Ohio, and Ohio State University.

The findings could have implications for people with muscular dystrophies and other muscle diseases, as well as muscle damage due to other illnesses, injury or aging.

MDA Grantees Prove Gene Therapy Grows Muscle Strength & Size in Primates

Four macaque monkeys that received injections of genes for a protein called follistatin into upper leg muscles experienced pronounced and durable increases in muscle size and strength and no adverse effects, the Muscular Dystrophy Association (MDA) announced today. The findings could have implications particularly for injured and aging people worldwide; and for tens of millions experiencing muscle loss associated with cancer, AIDs and muscle diseases.

LGMD Research: Gene Decreases Severity

New research has shown that a change in a gene not previously connected to type 2C limb-girdle muscular dystrophy (LGMD2C) modifies the severity of the disease in mice and is likely to do the same in people with this and perhaps with related types of muscular dystrophy.

DMD Profile: The Weather Guy

Long before a freak blizzard ripped into town and left more than a foot of snow in its wake, the citizens of Crossville, Tenn., and region had been warned to prepare for a freezing onslaught.

Fully a week earlier, local meteorologist Steve Norris had taken a close look at his charts and data and determined, “Whoa! We’re in for a big one.”  A full-time radio broadcaster for Crossville radio station WIHG (“The Hog”), Norris got the word out to most of eastern Tennessee and also to a wider, multi-state area via TV stations and newspapers which routinely check his weather reports.

Still Fishing: BMD Profile

Thanks to a little help from a tablecloth, a scissor jack and his wife Mary, Curt Sweely is one heck of a fisherman.

Of course, Sweely has been one of the fishing-est fishermen on East Coast lakes for decades, with a boatload of trophies and titles to show for it. The only thing that’s changed is his approach.

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

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.

Exon Skipping Proves Effective in Mice with a Severe DMD-Like Disease




Stephen Wilton

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