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 clinical trials. Why does it take so long?

MDA has awarded $750,000 to Summit Corporation PLC for development and testing of SMT C1100, the company's experimental drug for treatment ofDuchenne muscular dystrophy (DMD). Summit is an Oxford, United Kingdom, drug discovery company.

The award was made through MDA's Venture Philanthropy (MVP) arm, a part of MDA's translational research program.

A December 2011 podcast from Nationwide Children's Hospital in Columbus, Ohio, explores how an inhibitor of a protein called NF-kappa B has been beneficial in a mouse model of Duchenne muscular dystrophy (DMD) and is now being developed as a potential DMD treatment.

The podcast is part of a Nationwide Children's series called "This Month in Muscular Dystrophy."

Two pharmaceutical companies recently announced they are expanding their development and testing of exon-skipping drugs for Duchenne muscular dystrophy (DMD).

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 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.

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, there are people like you and me here!’ He was just beaming.”

Exon skipping is a strategy currently being developed for Duchenne muscular dystrophy (although it may have application to other genetic diseases down the line) in which sections of genetic code are “skipped,” allowing the creation of partially functional dystrophin, the muscle protein missing in DMD.