Researchers, patients meet in Clearwater to tackle a complex muscular dystrophy

For the next three days, Florida will be the center of the scientific world for patients and researchers concerned about myotonic dystrophy, a disease that is the most prevalent, yet one of the least recognized, types of muscular dystrophy.

More than 200 scientists, physicians and health care professionals are sharing their discoveries at the Sheraton Sand Key Resort in Clearwater during the International Myotonic Dystrophy Consortium, a scientific gathering that has occurred every two years since the inaugural meeting in Paris in 1997.

“Patients from all over the globe will have a unique opportunity to meet and hear firsthand about the progress researchers are making toward viable treatments,” said Tetsuo Ashizawa, M.D., the founder of the consortium and executive director of the University of Florida’s McKnight Brain Institute.

Myotonic dystrophy strikes adults, children and even newborn babies with progressive muscle degeneration and weakness. It also affects the heart, central nervous system, gastrointestinal tract, endocrine organs and eyes. As a muscular dystrophy, it is unique because it prevents skeletal muscles from relaxing once they contract.

“We are an informal group of clinicians and research scientists who have a common interest in understanding the molecular events that result in this neuromuscular disease, and using this information to generate effective treatment strategies,” said event co-chairman Maurice Swanson, Ph.D., a professor of molecular genetics and microbiology at UF’s College of Medicine and a member of the UF Genetics Institute. “The scientists strive to translate their work into layman’s terms and help patients and their families understand the state of the research.”

Patient involvement is coordinated by the Myotonic Dystrophy Foundation, the event sponsor.

“On Sunday, the meeting with patients will continue with more than 400 participants, making it the largest patient gathering for this disorder in history, anywhere in the world,” Ashizawa said.

In addition to Clearwater and Paris, the consortium has met in Raleigh, N.C.; Kyoto, Japan; Glasgow, Scotland; Quebec City, Canada; Milan, Italy; and Wurzburg, Germany.