Small molecules that induce or reverse effects of the type 1 myotonic dystrophy mutation in cells in the lab are likely to speed research
posted on July 9, 2013 - 11:01am
Matthew Disney is a current and former MDA grantee at the Scripps Research Institute in Jupiter, Fla. Disney's current MDA grant is focused on targeting toxic RNA in type 2 myotonic dystrophy (MMD2, or DM2).
*Many people interviewed for this article asked not to be identified to protect the privacy of affected family members.
“Everybody knows the word apathy,” says a California woman whose 25-year-old daughter’s type 1 myotonic dystrophy (MMD1, sometimes called DM1) was diagnosed just a few years ago. “People use the word loosely. I don’t think it does justice to the reality of this disease.”