University of Rochester

Adding a cell-penetrating peptide to a compound that reduces clump formation in cell nuclei increased its effectiveness in type 1 myotonic dystrophy research mice

posted on February 27, 2013 - 3:51pm
Scientists at the biopharmaceutical company Genzyme, working with mice, say they have modified and improved an existing experimental strategy to treat type 1 myotonic muscular dystrophy (MMD1, also known as DM1).

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.