Researchers funded in part by MDA found that a single blood-vessel injection of myotubularin genes markedly improved muscle abnormalities and survival in mice and dogs
Update (Feb. 3, 2014): The video from the University of Washington has been edited, and the link has been updated to reflect this.
Update (Jan. 27, 2014): MDA also supported Alan Beggs at Harvard University on this study. We regret not including this information intially.
A $1.2 million MDA grant will help move an experimental cell-penetrating treatment for myotubular myopathy toward human testing
The Muscular Dystrophy Association has awarded $1,195,762 over two years to biotechnology company Valerion Therapeutics (formerly 4s3 Bioscience) for development of a treatment for myotubular myopathy (MTM).
Mice with a disorder mimicking myotubular myopathy showed improvements in muscle strength and function in response to a myotubularin-based enzyme replacement therapy
Update (Jan. 10, 2013): This story has been updated with additional information about MDA funding of this and related research projects.
When Patrick and Sarah Foye of Pine Brook, N.J., had their baby boy, Adam, in 2001, there was little reason to believe at first that anything was wrong. "Adam had a normal birth," Sarah says, but things didn't go as well as expected in the months that followed.
Patrick is a physician specializing in rehabilitation, and Sarah is an occupational therapist, so they were quick to note that something...