Research

Researchers at the University of Virginia recently published a paper describing a biological pathway they believe may be responsible for muscle degeneration in DM1.

Professor Darren Monckton of the University of Glasgow in Scotland has been fascinated by genetics since very early in his career. Today he heads up a major genetic disease research group focused largely on understanding the relationship between DM's underlying genetics and symptoms in families and individuals.

The MDF 3.0 Roadmap is available for your review. MDF has pledged our resources to a number of significant aimed at accelerating Care and a Cure for the next three years.

French geneticist Dr. Geneviève Gourdon has developed mouse models to study the mechanisms responsible for DM's genetic repeat, and possible ways to combat the damage this repeat causes.

Researchers at the University of Florida, led by Dr. Tetsuo Ashizawa, recently published a study in which they developed a strategy for DM1 stem cell therapy involving gene modification.

As a clinician and researcher, Dr. Benedikt Schoser of Ludwig-Maximilians University of Munich, Germany, is focused on how research findings can be translated into improved patient care - and how patient concerns can help guide researchers to new areas of interest.

To support myotonic dystrophy drug development efforts, MDF held a science workshop in September 2014 at our annual conference. The workshop brought together more than 50 DM researchers and industry representatives from around the world to review where we are in the development of clinical trial endpoint measures for DM.