Parents Join Together Across Borders to Cure Rare Disease

If it is true that it takes a village to raise a child, what then does it take to save the life of a child or many suffering from a rare, and seemingly incurable disease? What it requires is seismic intervention on the part of a global community of parents who refuse to sit idly by and watch their children succumb to a deadly disease. Parents Kiko and Maria from Ares, Spain exemplify what it means to have a warrior spirit that stems from radical hope. With the help of their community, they raised over $250,000 USD in honor of their son, Adri, who is affected by Hunter syndrome — a rare, progressive disease that typically robs children of their lives before the age of fifteen. 

They chose to collaborate with Project Alive, a US-based nonprofit, because they “believe that [the organization] is clearly in line with our hopes and we are confident that one day, together, we will make mucopolysaccharidosis type II simply difficult to pronounce, but no longer a hopeless diagnosis.”

This is the largest one-time donation the US organization has received. Regarding the donation, President Kim Stephens commented,

“We are so grateful to Una Cura Para Adri for its generous donation. Project Alive was founded on a dream of starting a gene therapy trial for our boys — all of our boys, whether in the US, Spain, or anywhere in the world. And this donation will bring us closer to making that a reality.”

In July 2021, as the16th International Symposium on MPS and Related Diseases wrapped up in Barcelona, Spain, parents from across the globe were working towards a related goal: fundraising for a cure for children affected by MPS II, also known as Hunter syndrome. International collaboration of scientists and researchers is imperative, especially for those investigating rare diseases like Hunter syndrome, as data on rare diseases like Hunter syndrome is scarce–a fact known all too well by parents of children suffering from these rare diseases. “As we just recently saw demonstrated at the MPS International Symposium,” said Project Alive President Kim Stephens, “worldwide collaboration and cooperation can lead to the innovation and breakthroughs we need in Hunter syndrome. Together we can discover the treatments and cures to help our kids live long and healthy lives.” 

Project Alive is a parent-led nonprofit organization committed to finding and funding a cure for Hunter syndrome (also known as Mucopolysaccharidosis or MPS II) through research and advocacy. The organization funds promising curative research, assists researchers and industry with designing research studies for our community, and advocates for the most effective and efficient system of clinical research, evaluation, and approval. To date, the organization has raised nearly $2.5M towards its goal of funding a gene therapy trial to cure Hunter syndrome. 

Contact: Kim Stephens, President of Project Alive, kim.stephens@projectalive.org