Research

posted on February 26, 2015 - 11:30am
MDA research grantee Charles Gersbach, assistant professor of biomedical engineering at Duke University, and colleagues, recently announced an advance in gene modification that could turn out to be a game-changer for boys and young men with Duchenne muscular dystrophy (DMD). The team's results were published Feb.
posted on February 13, 2015 - 11:50am
The National Institute of Nursing Research (NINR) at the National Institutes of Health in Bethesda, Md., is conducting a study to determine if an antioxidant drug that has been approved by the U.S. Food and Drug Administration for other indications can reduce the severity of some symptoms in people with central core disease (CCD), a genetic disease caused by defects in a structure known as...
posted on February 2, 2015 - 12:04pm
Development of treatments for Duchenne muscular dystrophy (DMD) continues to advance. Many of the new investigational drugs are potentially applicable to all DMD patients, while a few target those with specific mutations in the dystrophin gene. Fighting fibrosis
posted on January 13, 2015 - 3:36pm
Boys with Duchenne muscular dystrophy (DMD) caused by specific genetic mutations who were treated with the experimental drug eteplirsen for 168 weeks (3.2 years) continued to show an advantage in the distance they were able to walk compared to those treated with the drug for only 144 weeks (2.8 years), although all boys had declined in walking distance since the start of this phase 2b trial of...
posted on January 7, 2015 - 12:06pm
Ever wondered how someone with a neuromuscular disorder in his or her family might contribute to research efforts in this field?
posted on January 5, 2015 - 9:25am
When Lizzie Chamberlain of Jacksonville, Ill., was 2 years old, her physical development relative to other children began to plateau — and then decline. Lizzie received a diagnosis of type 3 spinal muscular atrophy (SMA). 
posted on January 5, 2015 - 9:23am
Considering the title of this column, From Where I Sit, I’m surprised more people have not written about butts. After all, for those of us wheeling around at a seated height — I live with type 2 spinal muscular atrophy (SMA) — other people’s butts are often what we see from where we sit. 
posted on January 5, 2015 - 9:21am
Even a quick search online will turn up all manner of white papers, analyses and studies that identify a higher risk of anxiety and depression among individuals living with chronic disease and physical disabilities — such as those associated with many types of neuromuscular disease.