Rare Daily Staff
Digital health data platform provider Backpack Health said it has launched a research study with the nonprofit research and advocacy group Project Alive to advance the understanding of Hunter syndrome.
Hunter syndrome, also known as MPS II, is a genetic metabolic condition or lysosomal storage disorder that affects one in 100,000 to one in 170,000 individuals and is almost exclusively found in males. Individuals affected with a severe form of the condition begin to lose basic functional skills between the ages of two and four and have a shortened life expectancy.
Under the collaboration, Project Alive will use patient-reported data from Backpack Health’s Hunter Syndrome International Patient Registry to find potential associations between approved and experimental therapies and toileting skills among patients with neurocognitive and attenuated forms of Hunter syndrome.
Jim Cavan, founder and CEO of Backpack Health, said that patients and caregivers often identify the impact of a rare disease early, even before the clinician or care team.
“In the rare disease community, we often have to wait until researchers come to us with a possible treatment,” said Kim Stephens, president of Project Alive. “In this partnership with Backpack Health, the Hunter syndrome community is being more proactive and actively helping researchers collect meaningful data and find a cure.”
Backpack Health’s customized health data management platform for Hunter syndrome helps users control their health journey with a secure centralized location for tracking, storing, and sharing health information. Backpack Health users who are part of the Hunter syndrome community can now opt-in to participate in quarterly surveys. The next survey launched on November 19.
“Giving patients more control over their own health data will change the way we approach research studies,” said Christian Hendriksz, an advisor to Project Alive and a professor of pediatrics and child health at the University of Pretoria where he’s been involved in multiple clinical trials for MPS II. “With more patients enrolled in a registry, we can begin making connections and uncover insights that may have previously gone unnoticed.”
Photo: Kim Stephens, president of Project Alive