Reducing dynamin 2 protein levels benefited mice with a disorder mimicking human myobutular myopathy; the strategy could have implications for MTM and additional diseases

posted on April 15, 2014 - 10:04am
A French research team has found that reducing levels of a protein called dynamin 2 has potential as a strategy to treat myotubular myopathy (MTM), a form of centronuclear myopathy (CNM), and that it could have implications for other nerve and muscle disorders as well.

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

posted on January 22, 2014 - 12:03pm
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

posted on May 30, 2013 - 5:00am
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

posted on January 10, 2013 - 3:25pm
Update (Jan. 10, 2013): This story has been updated with additional information about MDA funding of this and related research projects.

A range of outcomes

posted on September 1, 2007 - 10:07am
QUEST Vol. 14, No. 5
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...