Project Alive Paves the Way for Gene Therapy Research That Could Save Children With Hunter Syndrome

Gene therapy is on the rise, and it just might be exactly what many children suffering from genetic disorders need to save their lives.

Today reported that an amazing organization called Project Alive has raised half the funds needed for Ohio Nationwide Children’s Hospital researchers to take next steps– that’s a $743,000 dollar check working towards the 1.4 million dollar goal. This will eventually help hundreds of children with rare diseases, and specifically develop a treatment to save the lives of children with Hunter syndrome.
Hunter syndrome, also called Mucopolysaccharidosis Type II (MPS II), is a rare, inherited disease that slowly regresses a child’s ability to function. First, they may show slightly delayed development, and then they will start to lose skills. The ability to walk, talk, eat, and even breathe begins to deteriorate. While medical intervention helps some patients live longer lives, many others pass away early on. To learn more about this rare disease, click here.

When the Muedder family learned their 2-year-old son, Finn, had Hunter syndrome, they were absolutely devastated. As his mother Allison puts it, it was a death sentence. They quickly learned there was no cure, but there was a treatment. It wouldn’t fix the problem, but it could prolong Finn’s life. They proceeded.

Shortly after diagnosis, the Muedders went on a mission to find something to help save their little boy. After failing to get Finn into a potentially positive clinical trial, Allison found the nonprofit Project Alive who was in the midst of raising funds for gene therapy– a treatment that they learned could save their son.

Project Alive began as a dream of Melissa Hogan’s, who started fighting for a cure in 2014, when her son, Case, was diagnosed with Hunter syndrome. Hogan, who came from a law background, recognized that gene therapy would be more effective than current treatments, both in terms of the child’s health and cost feasibility.

With half the funds raised the researchers have developed a therapy for the disease, but now they need the rest of the funds to develop vectors– the mechanism that deliver the treatment to the cell– to complete the task and begin testing it. Project Alive has brought so many families with Hunter syndrome together for a common mission, and they all can just taste what reaching that final goal will feel like.

There’s no time to relax– it’s time for more fighting. They need to raise another $700,000 in the first half this year for the research to go on. Once they secure the funds, the FDA has already approved the testing, and depending on the results, they can be off to the races to help save 2,000 children around the world affected by the disease.

Help make a change the lives of Finn and Case, as well as hundreds of other children right now here!


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