Drugging the undruggable: Yale finds treatment path for muscular dystrophy

September 11, 2020

A research program led by Dr. Anton Bennett has identified a possible treatment for Duchenne muscular dystrophy (DMD), a rare genetic disease for which there is currently no cure or treatment, by targeting MKP5 phosphatase that had been considered “undruggable”.  YCMD scientists including Laura Abriola, Jim Herrington and Pete Gareiss supported assay development and high-throughput screening of diversity libraries for this program, which identified a drug-like compound that inhibited the enzyme by binding to an allosteric site. The findings have implications well beyond muscular dystrophy, and we are honored to support this important work.

Please see the complete YaleNews coverage by Fred Mamoun here.