Foundation "Project Alive" Raises Over $100,000 on Giving Tuesday
Families Are Making A Cure A Reality for the Rare Disease Hunter Syndrome / MPS II
By: Project Alive
Hunter Syndrome is a rare, genetic condition that affects approximately 2,000 people worldwide, almost exclusively young boys, causing progressive loss of physical, and in most cases, mental function. The average life span for someone with Hunter Syndrome is in the early teens.
The funds from Giving Tuesday will go directly towards the manufacture of a gene therapy vector for a Phase I clinical trial, set to begin in 2018 if Project Alive secures the needed funding. Gene therapy has been shown to be a cure in the mouse model of the disease. Currently, the only FDA-approved treatment option for Hunter Syndrome is a weekly 4-hour infusion that merely stabilizes some of the disease's physical symptoms. But that option is at a cost to the health care system of approximately $400,000 per patient, per year, and doesn't even prevent the progressive neurological dementia-like decline that ultimately takes the lives of the children affected.
"Project Alive is about restoring the hopes of families for their affected children to live and grow up," notes Project Alive President Melissa Hogan of Thompson's Station, Tennessee. "We are so grateful that friends and families, members of the public, as well as some celebrities and larger foundations, have come alongside those efforts. As a Hunter Syndrome parent myself, I vividly remember those dashed hopes when my own son Case was diagnosed in 2009."
To help spread the word, Project Alive has been releasing a mini-documentary series featuring families of children with Hunter Syndrome, sharing the symptoms, diagnosis, and prognosis of this devastating disease and how the public can help fund research toward a cure. Produced by Caravan, a Charlotte, NC - based production agency led by the father of a child with Hunter Syndrome and boasting clients such as HBO, Pixar, and Coca-Cola, the series offers viewers a glimpse into the lives of families facing this devastating diagnosis. The series has been featured in a film festival and received several other honors. The series can be viewed at ProjectAlive.org or on GoFundMe at www.gofundme.com/
Check out one of the documentary episodes on youtube at https://www.youtube.com/