Project Alive

Exhibiting Partner

We exist to find a cure for Hunter syndrome through research and advocacy, and providing education and resources to the Hunter syndrome community. We are here to give a voice to the families and children touched by Hunter syndrome.

Project Alive is a powerful voice for children and adults with Hunter syndrome, bringing together families and advocates with researchers, industry, and regulators. 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. Through its innovative campaigns and grassroots efforts, Project Alive has made significant advances in public awareness about Hunter syndrome and its symptoms, the need for early diagnosis, and available treatments and clinical trials.

Our promise as a community:

We welcome and embrace those families who are newly diagnosed. We hold close those families who are journeying alongside us. And we support and comfort those families whose children have passed – never forgetting the impact of their lives and the joy they brought us.

In 2019 Project Alive made a conscious effort to expand our mission beyond one gene therapy trial. Recognizing the significant interest in Hunter syndrome from the pharmaceutical industry, we set out to partner with everyone interested in helping our boys to make sure each clinical trial is successful. This allows us to advocate for every child with Hunter syndrome and ensure the best possible outcome for each family.

We are also leading the research in Hunter syndrome by being proactive to fill the gaps in the literature and create our own outcome measure for clinical trials – The Toileting Abilities Study. Project Alive has brought together the leading physicians and researchers to update the MPS II Multidisciplinary Guidelines, so each child diagnosed with Hunter syndrome will get the latest and best care. Publication of the guidelines is slated for 2023.

Project Alive is also actively working on pushing Newborn Screening. MPS II has been nominated for the US federal Recommended Uniform Screening Panel (RUSP) currently before an advisory committee. We know we must be ready to serve those newly diagnosed families and give them the best possible information to help them on their journey.

And with the help of the Hunter syndrome community and all our wonderful donors, we have moved forward with the gene therapy trial that we dreamed about back when Project Alive first formed. With the tremendous fundraising efforts of the community, we have completed the research to successfully get the designation from the FDA of Investigational New Drug (IND). We are pleased to share we have moved the trial to UNC, Chapel Hill, where we can collaborate closely with Dr. Joe Muenzer to move forward.

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