MBNL

A small-molecule compound counteracts some of the effects of abnormal genetic instructions in MMD1

posted on November 3, 2009 - 10:00pm
  A compound that has the potential to be refined and modified into a treatment for type 1 myotonic dystrophy (MMD1, or DM1) has been identified by researchers at the University of Oregon in Eugene, and the University of Rochester (N.Y.) School of Medicine and Dentistry.

Compound frees a crucial protein from a cellular trap in mice with MMD1; treatment prospects 'bright'

posted on July 17, 2009 - 4:03pm
Researchers at the University of Rochester (N.Y.) Wellstone Muscular Dystrophy Cooperative Research Center have identified a compound that has the potential to be developed into a treatment for type 1 myotonic dystrophy (MMD1, or DM1). The compound, dubbed CAG25, is an "antisense oligonucleotide," a type of construct that's been used to block disease-causing genetic instructions in laboratory...