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.
Dr. Tetsuo Ashizawa, MD, has focused his career on the search for DM treatments and care for those affected. His multi-disciplinary, patient-centric approach to care moves DM research out of the lab and into his clinic at the University of Florida.
Dr. John Vissing and his colleagues at the University of Copenhagen recently tracked a group of 68 adults with myotonic dystrophy type 1 (DM1), measuring their endocrine function change over 8 years.