Muscular Dystrophies

MDA Awards $13.7 Million in Research Grants

The Muscular Dystrophy Association has awarded 40 research grants totaling $13.7 million to advance the understanding of disease processes and uncover new strategies for treatments and cures of muscular dystrophy and the more than 40 other diseases in the Association's program.

The new grants were recommended by MDA's Scientific and Medical Advisory Committees and approved by MDA's Board of Directors at its July 2011 meeting.

Global CMD1C/LGMD2I Registry Opens

A new registry has been launched for people with conditions caused by mutations in the gene for fukutin-related protein (FKRP). This includes people with the type 2I form of limb-girdle muscular dystrophy (LGMD) and the type 1C form of congenital muscular dystrophy (CMD) and, in rare instances, the congenital muscular dystrophies muscle-eye-brain disease and Walker-Warburg syndrome.

Families and Experts Gather at 2011 BMD Conference

Community and empowerment were the themes of the third annual Becker Muscular Dystrophy (BMD) conference held at Cedars-Sinai Medical Center in Los Angeles on August 13, 2011.

Some 150 in-person attendees, and at least that many online attenders, engaged physicians, researchers, physical therapists and other BMD experts on a variety of topics, including the latest advances in research, medical management, physical therapy and clinical trials.

Return Me to My Special Place

Artist: 

Gene transformed his love of nature into intricately detailed wood carvings of wildlife.  For many years he had a gallery, "Waterfowl by Gene," in Stockton and was active in the city's Arts Council.

Prarie Road Homestead

Artist: 

Connie has been actively photographing landscapes and rural scenes for over 10 years and has won several awards for her outstanding photographic work.  This photo was taken along Prairie Road near Mountain Home, Idaho.  Descendants of the family that built this home still live in the area.

Bees On Sunflower

Artist: 

Connie has had many of her photos published by local media and has also won several awards for her outstanding work.  She attended Central Washington University for two years studying liberal arts.  Connie enjoys growing sunflowers, collecting rubber stamps and postcards.

DMD: Phase 2 Exon-Skipping Trial Launched

Updated: View MDA's video about the launch of the eteplirsen trial on YouTube.

Connie Prater

Location

Boise, ID

Connie has had many of her photos published by local media and also has won several awards for her outstanding work. She attended Central Washington University for two years, studying liberal arts. Connie enjoys growing sunflowers, and collecting rubber stamps and postcards.

Full name: 
Connie Prater
Artist: 
Connie Prater
Disease: 
Muscular Dystrophies
Medium: 
Photograph

Gene Ashby

Location

Stockton, CA

Gene transformed his love of nature into intricately detailed wood carvings of wildlife. For many years he had a gallery named Waterfowl by Gene in Stockton and was active in the city’s Arts Council.

Full name: 
Gene Ashby
Artist: 
Gene Ashby
Disease: 
Muscular Dystrophies
Medium: 
Hand Carved Wood

Eteplirsen (AVI-4658) Boosts Dystrophin Production in DMD

Complete and extremely encouraging findings from a phase 1b-2 trial of eteplirsen (AVI-4658), an exon skipping drug in development to treat a portion of the Duchenne muscular dystrophy (DMD) population, show the compound is safe and well-tolerated, and that it can significantly increase production of the needed dystrophin protein in recipients without eliciting an unwanted immune response.

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