MDF is pleased to welcome Laura Ranum to its Scientific Advisory Committee (SAC). Dr. Ranum, who joined the SAC in summer 2015, is an internationally known investigator of disorders that result from repeat expansion mutations, such as those that cause type 1 and type 2 myotonic dystrophy (DM1 and DM2).
The Myotonic Dystrophy Foundation partnered with Silicon Valley Research Group to develop a survey to better understand how people with DM weigh the benefits of new treatments again the risks.
MDF is pleased to welcome Tom Cooper, MD, to our Scientific Advisory Committee (SAC). Dr. Cooper, who joined the SAC in summer 2015, is a renowned myotonic dystrophy (DM) investigator whose laboratory has made major contributions to understanding the molecular pathogenesis of the disease and pointing the way toward rational therapeutic development.
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.