Hansell Stedman, a surgeon who had two brothers with Duchenne muscular dystrophy, aims to translate lessons from crush injuries into improvements in gene therapy
You don’t have to look far to figure out how Hansell Stedman got interested in Duchenne muscular dystrophy (DMD). His older brother, Holt, and younger brother, Roland, had the disease.
Four children not making any acid maltase enzyme tolerated enzyme replacement therapy when also treated with immunosuppressants
Editor's note 2/2/12: This story was updated to reflect the award of a new MDA grant to Eric Sjoberg at Amicus Therapeutics.
Drugs that suppress the immune system can successfully prevent or reverse rejection of enzyme replacement therapy in Pompe disease (acid maltase deficiency), a new study has shown.
Biologist Denis Guttridge discusses his team's work developing an NF-kappa B blocker as a potential treatment for Duchenne MD
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."
Research items about Friedreich's ataxia, myasthenia gravis, mitochondrial myopathies, type 1 myotonic dystrophy, gene therapy and gene modification
Edison drugs target FA, mitochondrial diseases
Carlie Brinker of Millersburg, Ohio, tries to walk to the mailbox and back a couple of times a day and to her sister’s house, half a mile away, if the weather is good.
Knowing if you're on the right course with corticosteroids
"Since being on prednisone, I’ve been up and down with my weight and up and down with the milligram dosage,” says Carlie Brinker. “I’m 19, and I’ve been on prednisone for 11 years.”
Usually, inflammation of tissue occurs with an injury or an infection, but it's also an important part of certain neuromuscular diseases.