Causes: Health, Specifically Named Diseases Research

Mission: Project Alive (formerly Saving Case & Friends, Inc. ) is a 501(c)(3) nonprofit Hunter Syndrome research & advocacy foundation. It exists to find and fund a cure for Hunter Syndrome / Mucopolysaccharidosis II through research, fundraising, and advocacy. Project Alive raises funds and then distributes them to research programs focused on Hunter Syndrome, as well as assisting third parties in designing research and clinical programs most likely to be successful in treating Hunter Syndrome. Project Alive also advocates with regulatory and legislative bodies, as well as industry, for policies supporting the most effective and efficient clinical research programs and compassionate use / expanded access protocols.

Results: Project Alive has been funding gene therapy preclinical research, along with a coalition of Hunter Syndrome foundations, for several years. The founders of Project Alive recruited researchers and collaborated with them during several years of preclinical research, culminating with the preparation for a first-in-human clinical trial expected to begin in 2018.Currently, Project Alive is raising the remaining funds to support the Phase I/II clinical trial. Project Alive has released a mini-documentary series on Hunter Syndrome families, supported the community through personal, regulatory, and industry advocating, and served as a resource for various Hunter Syndrome clinical research teams. Members of our leadership have also served on several panels at FDA and NIH workshops, authored expert pieces related to Hunter Syndrome research and clinical trials, and submitted comments on numerous public dockets related to rare disease research, patient advocacy, and regulatory matters.

Target demographics: families impacted by Hunter Syndrome

Geographic areas served: worldwide

Programs: advocacy, fundraising, and research support