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.
At MDF's annual conference in September we heard from four of the newest MDF-funded Fellows, and from three pharmaceutical companies involved in early stage drug development for myotonic dystrophy.
Researchers from Ionis Pharmaceuticals collaborated with university teams from across North America to test out what could be the next generation of antisense drugs targeting myotonic dystrophy type 1 (DM1). As our community knows, the potential therapy currently in a phase I/II clinical trial, IONIS-DMPKRx, is also an antisense drug.
The Clinical Trials Transformation Initiative (CTTI), a public-private partnership between Duke University and the FDA focused on increasing the efficiency of clinical trials, has released a detailed series of recommended best practices for interactions between patient groups and clinical trial sponsors.