Dr. Haiyan Fu from the University of North Carolina School of Medicine joined Project Alive Vice President Mario Estevez, film producer Joey Howell and Florida Gulf Coast University students for a panel discussion and screen of the documentary Boys with Bigger Hearts at FGCU Oct. 18.
FGCU Professor Courtney Dwyer-Satkoski called it an “evening of research, advocacy, storytelling, and hope.” Her students formed Hunting for a Cure in 2016 to help in the fight against Hunter syndrome.
Project Alive parents from Florida to California have worked closely with researchers to develop an effective gene therapy product to treat individuals with MPS II. Dr. Kim McBride, the clinical principal investigator of the project, has been working for two decades in collaboration with Dr. Fu at Nationwide Children’s Hospital and Dr. Joseph Muenzer at University of North Carolina at Chapel Hill on the research.
In 2013, Project Alive began working with Dr. Fu to see if her previous preclinical gene therapy research for a similar disease, Sanfilippo Syndrome (MPS III), could be developed in Hunter syndrome. It is a symbiotic relationship between a researcher who knew the science and parents most familiar with the disease, which accelerated the then ongoing collaborative research project of Drs. Fu and Muenzer.