Our Mission is to raise awareness and funds for Sanfilippo research that will lead to a cure, and to instill and sustain hope for families impacted by the disease.
Sanfilippo Syndrome, otherwise known as Mucopolysaccharidosis IIIA (MPS IIIA), is a rare and terminal genetic disease that affects children. It has no cure or treatment. Children develop typically for the first couple years of life, then they slowly lose all the skills they have gained, including the ability to talk, walk, and even swallow, eventually leading to death in the second decade of life. As children regress, irreversible damage occurs to the brain and body resulting in loss of mental function, extreme hyperactivity, sleeplessness, seizures, and difficult behaviors.
Promising research is occurring around the world, some of it right here in Columbus, Ohio where researchers at Nationwide Children's Hospital have created a gene therapy treatment that could help treat this awful disease. The human clinical trial began in the spring of 2016 and is ongoing. Research continues around the world, including the start of several other clinical trials. We are in a race against time to stop this awful disease. Our mission isn’t over until ALL children with Sanfilippo Syndrome have a cure or treatment. Reagan's Hope: A Cure for Sanfilippo, cannot be accomplished without your help. Please help be part of the cure!
Our lives became complete on March 1, 2012 when our beautiful baby girl, Reagan, came into this world. Two years later our world was then turned upside down when Reagan was diagnosed with Sanfilippo Syndrome. We were told it was terminal with no cure or treatment, and we should take her home and enjoy the short time we had. As parents, we refused to accept this fate for our daughter, so we created Reagan’s Hope, A Cure for Sanfilippo Foundation in May of 2014.
Living with Sanfilippo Syndrome: Reagan’s Story, created in 2014 by Nationwide Children’s Hospital as they moved towards a gene therapy clinical trial. The trial started in the spring of 2016 and is on-going.