oligonucleotide

Antisense compound given to mice with a disorder resembling type 1 myotonic dystrophy safely corrected molecular defects and myotonia for a year

posted on August 2, 2012 - 11:01am
An experimental treatment for type 1 myotonic muscular dystrophy (MMD1, or DM1) has corrected several aspects of the disease in an MMD1 mouse model. A year after treatment with an experimental antisense oligonucleotide dubbed ASO 445236 ended, positive effects remained apparent in the mice.

An MDA-sponsored meeting explored progress in five key therapeutic strategies under development for neuromuscular diseases

posted on March 18, 2011 - 3:30pm
Moving therapeutic strategies from the laboratory to clinical trials and ultimately to the market as treatments was the theme of the MDA National Scientific Conference held March 13-16, 2011, in Las Vegas. Some 300 people attended the conference, the first in a planned series of such MDA-sponsored meetings that will emphasize new research and current medical care. The majority of presenters and...