The shorter-than-normal dystrophin protein produced via an experimental exon-skipping drug allows proper localization of proteins at the muscle-fiber membrane
Shortened versions of the muscle protein dystrophin — produced by skipping a section of genetic instructions called exon 51 — appear to be functional, says a new report from the United Kingdom.
MDA has awarded 38 grants totaling more than $12 million to support research into general muscle health and more than 15 neuromuscular diseases in its program
The Muscular Dystrophy Association has awarded 38 new grants totaling more than $12 million to fund research projects focused on its continuing mission to uncover the causes of, and develop therapies for, the more than 40 neuromuscular diseases in its program.
MDA's Board of Directors reviewed and approved the new grants based on recommendations from the Association's Scientific and Medical...
MDA's $1 million grant will help fund the development of TVN-102, which attracts the muscle protein utrophin to the muscle-fiber membrane
The pace of research can seem unreasonably slow; here are a few reasons why
John Porter from the National Institutes of Health likes to start talks by noting, “It’s a great time to be a mouse with a neuromuscular disease.” Exciting research results are regularly reported, where a treatment appears to cure one neuromuscular disease or another in a mouse — yet there are few treatments available today for people with any of these diseases, and only a few treatments in human...
Researchers at six centers are studying possible cardiac benefits of dietary supplement coenzyme Q10 and cardiovascular drug lisinopril in three MD forms
Researchers at five U.S. and one Canadian center are conducting a clinical trial of the medications coenzyme Q10 and lisinopril to determine their possible beneficial effects on heart function in Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD) and five forms of limb-girdle muscular dystrophy (LGMD).
A new, NIH-supported translational research center will focus on exon skipping as a treatment for MD
A Center for Research Translation of Systemic Exon Skipping in Muscular Dystrophy has been established by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), part of the U.S. National Institutes of Health.
Quest shares lessons from parents who have successfully helped their children with muscle diseases grow into self-reliant adults
When Vance Taylor was a boy, he didn’t know any adult he could look to and say, “There’s somebody like me.”
His mother, Morena Noyes, recalls the first time she took Vance and his sister Kathy — both of whom have limb-girdle muscular dystrophy — to MDA summer camp.
“We were still in the parking lot, in our Astro van,” Noyes says. “Vance looked, and then he turned to his sister and said, ‘Kathy,...