Gregory Nemunaitis, John Nemunaitis and colleagues from the Mary Crowley Cancer Research Centers in Texas have reported the first and only example of a single HIBM patient treated by intravenous infusion of a wild type GNE gene lipoplex. Read more about it here.
Dr. Paul Martin and colleagues at Nationwide Childrens Hospital in Ohio have been exploring a novel approach to treating GNE myopathy using a bicistronic vector technology. Read more about it here.
Professor Stella Mitrani-Rosenbaum from Hadassah University Medical Center is a leading international expert in GNE Myopathy and has published extensively on the topic. Read more about it here.