Muscular Dystrophies

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.

DMD: Daily, Weekly Prednisone Treatment Results About the Same

A one-year, MDA-supported study comparing a weekend-only prednisone treatment schedule with a daily prednisone schedule in boys with Duchenne muscular dystrophy (DMD) has found that the two treatment regimens provide about the same benefits and have approximately the same side-effect profile.

Research Briefs: FA, MG, MM, MMD1, gene therapy

Edison drugs target FA, mitochondrial diseases

Researchers Studying DMD-Affected Families

Belen Pappa, a graduate student in genetic counseling, is seeking participants for a survey-based study about psychosocial functioning in families in which a child has Duchenne muscular dystrophy (DMD).

Research Briefs: DMD, BMD Trials Update

U.S. trial of eteplirsen for Duchenne MD delayed

Signs and Symptoms

The pattern of muscle loss in BMD usually begins with the hips and pelvic area, the thighs and the shoulders. To compensate for weakening muscles, the person may walk with a waddling gait, walk on his toes or stick out the abdomen.

The rate of muscle degeneration varies a great deal from one person to another. Some men require wheelchairs by their 30s or later, while some manage for many years with minor aids, such as canes.

Research Briefs: Stem Cells

Stem cells are a hot topic these days in medicine, science and law, although the term has multiple meanings and it's easy to get confused.

In short, stem cells are cells at an early stage of development from which specialized cells, such as muscle or nerve cells, can develop (in other words, from which these specialized cells "stem").

Different kinds of stem cells are referred to as:

Research Briefs: The DMD/BMD-Affected Heart

In both Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), deterioration of the heart muscle, a condition known as cardiomyopathy, is a major cause of disability and death.

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