Working Toward a Cure
Doctors at Nationwide Children’s Hospital in Columbus, Ohio have conducted research that leads them to believe that a cure for MPS II has been discovered. Dr. Haiyan Fu, lead investigator, has conducted years of research and are relying on parent-led organizations to raise the funds needed to carry the drug development process through clinical trials. Human clinical trials involve administering the drug in a controlled environment to gather data on efficacy. This is usually done before making the drug available to all affected patients.
The total funds needed to start the FDA approved clinical trial are$2.5 million! The funds collected will cover expenses such as securing the facility where the clinical trials will be held, along with building of vectors that will be used to introduce the cure to our little SuperHeroes. Funds will also be used to cover medical costs, room and boarding costs, and miscellaneous costs for the patients who will be participating in the clinical trial.
As part of a conglomerate of parent-led nonprofit organizations across the country, a total of slightly over $2.4 million has been raised to date. Currently, the MPS II community are in need of $89,000 to see the elimination of MPS II finally come to fruition and the MPS SuperHero Foundation won’t give up until the goals are met!
Meet the Doctors working to save the lives of those affected by Hunter Syndrome (MPS II)
The Research Institute at Nationwide Children’s Hospital
700 Childrens Drive
Columbus, OH 43205
Douglas M. McCarty, PhD
Center for Gene Therapy