Ionis Pharmaceuticals is currently seeking trial participants to evaluate an experimental DM1 drug, IONIS-DMPKrx, that may help modify the disease rather than just treat the symptoms.
The results of a new MDF-funded study on the impact of congenital myotonic dystrophy are now available from Dr. Nicholas Johnson of the University of Utah and colleagues. The high frequency of social and cognitive issues in their findings underline the need for a multi-disciplinary approach to care.
Many common DM symptoms become more severe during pregnancy, and women with DM have higher than average miscarriage rates, according to a study commissioned by MDF that examined data from the Myotonic Dystrophy Family Registry and the National Registry for DM and FSHD. Read a summary of the findings written by Katharine Hagerman, PhD, Research Assistant at Stanford University.
Kathie Bishop, Ph.D.
San Diego, California
Member, Scientific Advisory Committee (SAC)
Myotonic Dystrophy Foundation (MDF)
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.