Logo of the GNEM Symposium Speaker Series, presented by The Neuromuscular Disease Foundation

First Online Presentation Expands Reach and Increases Impact

The Neuromuscular Disease Foundation (NDF) will launch its annual Symposium on GNE Myopathy Speaker Series on Friday, May 8th, albeit in a much different format than in previous years. The series is designed to educate both the patient and scientific communities on GNE Myopathy, an adult onset muscle-wasting disease. The goal is to feature specific updates from all NDF grant awardees for the 2020 cycle.

Friday’s presentation, “Exploring the Potential of Gene Editing as a Therapeutic in GNE Myopathy,” will be presented online by Dr. Monkol Lek, with Dr. Angela Lek, from the Lek Lab at Yale University. Other speakers in upcoming weeks will include presentations from investigators at the National Institutes of Health, Ohio State University, Mount St. Joseph University, University of California, Hadassah Medical Center in Israel and Japan’s National Center for Neurology & Psychiatry.

Lale’ Welsh, NDF’s CEO, explained the organization’s strategic pivot during these highly unusual times. “Our mission to update patients and other scientists must continue through the uncertainty of this devastating pandemic and its aftermath. Although some nonprofits have felt the need to cancel their events, we are fortunate that our outstanding presenters, our dedicated team and our highly committed participants have embraced this revised presentation format. Our program supports the rare disease patient community by enabling the world’s leading scientists studying GNE Myopathy to share information with the broadest audience possible.”

NDF’s Symposia continue to represent the preeminent gathering for GNEM scientists, physicians, patients, caregivers and industry personnel, creating historically unprecedented opportunities for scientific collaboration and patient education. In the past, NDF has helped make the attendance of patients from all over the world financially possible through scholarships.

“By taking these presentations online, NDF is now also creating a unique opportunity for patients who are homebound, or others who previously would have been unable to attend, to participate. Additionally, this pivot allows the cost savings generated to be used towards critical new programs including our upcoming telehealth services.” Ms. Welsh added, “The silver linings here are so many that this may very well become our new normal, moving forward.”

This first event will be followed by a series of presentations to be made by scientists and industry professionals, all with scientific endeavors being funded by NDF in its efforts to find curative treatments for GNEM. Registrants will have the opportunity to ask live questions at the conclusion of each presentation. A recorded version will be made available, free of charge, “on demand” on NDF’s website by anyone interested in learning more about GNE Myopathy.

About the Neuromuscular Disease Foundation: Founded in 2006, NDF is a registered 501(c)(3) public charity and the world’s leading foundation in funding research that supports the search for a cure for GNE Myopathy (also known as HIBM). Its mission is to enhance the quality of the lives of people living with GNE Myopathy through advocacy, education, outreach and the funding of critical research focused on treatments and a cure.