Project Alive and Action For Aidan partner with $400,000 towards scientific endeavors for Hunter syndrome

May 6, 2024

Project Alive, a national Hunter syndrome research and advocacy organization, has partnered with New Hampshire based non-profit, Action For Aidan, to collaborate on scientific research, publications, and other endeavors to benefit Hunter syndrome.

Hunter syndrome, also known as Mucopolysaccharidosis type II (or MPS II), is a life-limiting, progressively debilitating, x-linked lysosomal storage disorder. It affects roughly 2,000 people worldwide, the majority of which are children with life expectancies before reaching adulthood.

Both organizations share the mission to find a cure for Hunter syndrome. Currently, there is one FDA approved treatment for Hunter syndrome, but it does not treat the devastating neurological impacts of the disease and thus is not lifesaving. In the United States, three clinical trials are taking place with drugs intended to reach the brain. With the recent pharmaceutical involvement in the MPS II space, the two organizations have joined to support additional forms of science to improve the understanding of Hunter syndrome and its symptoms. With this hope, Project Alive and Action For Aidan have matched funds totaling $400,000 to dedicate to this pursuit.

To date, the organizations have committed to fully funding the first consensus meeting on behaviors in neurodegenerative MPS at the Muenzer MPS Treatment and Research Center located at UNC, Chapel Hill. This meeting will bring leading specialists together to discuss behavioral challenges in neurodegenerative MPS disorders, treatment options available for these challenges, and work to come to a consensus on best practices to manage behaviors. This meeting will result in a publication which can be used in clinics around the world. The funding will total roughly $230,000.

Project Alive and Action For Aidan have also committed to contributing $50,000 to the next MPS Master Class for Physicians and Nurse Practioners at the Muenzer MPS Treatment and Research Center. The Master Class aims to educate providers on these rare disorders to enhance clinical care experienced by patients and their families.

“We are grateful for this partnership with Action For Aidan as the organization is made up of a group of people truly dedicated to furthering science in Hunter syndrome. Project Alive is enthusiastic for the work already slated to be done this year, and we look forward to the continued work we will do together. There is so much research left untouched, and we hope to make an impact on science, but also on the daily lives of our families by enhancing the care they receive. Our community members all deserve comprehensive medical care. That is currently challenging with only a handful of experts in the field. It is wonderful to embark on spreading their knowledge across the globe,” states Kristin McKay, President and Executive Director of Project Alive.

To learn more about these organizations, visit www.projectalive.org and www.actionforaidan.org.