small-molecule strategy

Repligen soon will begin a first-ever human trial of a drug created specifically for spinal muscular atrophy;  FDA grants fast track designation

posted on May 20, 2011 - 11:13am
In a historic first, biotech company Repligen Corp., of Waltham, Mass., has received approval from the U.S. Food and Drug Administration (FDA) to begin a phase 1 clinical trial of the experimental drug RG3039 for spinal muscular atrophy (SMA).

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.