MDF hosted the Myotonic Dystrophy Patient-Centered Therapy Development Meeting in Washington, D.C. on September 17, 2015. The regulatory workshop featured speakers from academia, industry and the FDA.
Although up to 25% of people with myotonic dystrophy report that gastrointestinal symptoms are their most troubling issue, we still understand little about their cause. MDF Fellow Dr. Melissa Hinman at the University of Oregon is tackling this issue with Dr. Andy Berglund of the University of Florida using zebrafish models.
MDF will host a strategic workshop this fall for professionals from the Food and Drug Administration, academic research institutions and pharmaceutical companies interested in DM therapy development. This regulatory meeting will be the first of its kind for DM.
As potential therapies for myotonic dystrophy move out of the realm of hypothetical and into clinical testing, there is an urgent need to better understand what will be required from a regulatory standpoint to approve new therapies for this disease.
Since the discovery of the mutation causing DM1 was discovered in 1992, researchers have been trying to understand how the DNA mutation causes such a wide variety of symptoms. A new study may have found a genetic link between DM and Duchenne muscular dystrophy, potentially explaining the progressive muscle deterioration that occurs in adults with DM1