OUR PATH TO A CURE
Our Mission and Vision
Project Alive exists to find a cure for Hunter syndrome through research and advocacy, and provide education and resources to the Hunter syndrome community.
THE STEPS WE TAKE
Our Path To A Cure
Creating More Hopeful Futures
We aim to support families and patients affected by MPS II through education, providing connections to specialists, outreach in times of need, and programs supporting the needs of both patients and caregivers.
Advocating for Families
Advocacy is at the heart of our organization. In 2022, the long- term efforts advocacy efforts of the Hunter syndrome community were celebrated as MPS II was added to the Recommended Uniform Screen Panel (RUSP) for newborn screening.
Fundraising for a Cure
Project Alive was built on grassroots fundraising efforts. Families, supporters, and donors funded the development of a gene therapy vector which is currently set to launch as a gene therapy clinical trial at UNC Chapel Hill.
Supporting Research
Project Alive will launch a new research grants program in 2023. This will enable all researchers interested in studying MPS II the ability to access grant funds from Project Alive.
OUR PROMISE TO THE COMMUNITY
We Are Here For You
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.
PAVING THE WAY
How We Are Making An Impact
Changing The Process
Advocacy is at the heart of our organization. Project Alive board members and staff take part in many advocacy efforts. We work alongside EveryLife Foundation, NORD, and the National MPS Society in rare disease advocacy efforts in both the federal and state level. In 2022, the long- term efforts advocacy efforts of the Hunter syndrome community were celebrated as MPS II was added to the Recommended Uniform Screen Panel (RUSP) for newborn screening. This effort has now shifted to advocating on the state level to include MPS II on all newborn screening panels across the country. Project Alive has also set a new goal to advocate for equity in rare disease clinical trials.
Updating Researchers
Project Alive will launch a new research grants program in 2023. This will enable all researchers interested in studying MPS II the ability to access grant funds from Project Alive. As we have successfully seen the start of four clinical trials for MPS II, we hope to broaden our scope towards additional areas in which research is needed. Our goal is to use these research outcomes to better support specialists, educators, and current or future clinical trials. Researchers can find the application portal on our Research Grants page.
Screening For Newborns
Project Alive is 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.
Instrumental Collaborations
Project Alive was built on grassroots fundraising efforts. Families, supporters, and donors funded the development of a gene therapy vector which is currently set to launch as a gene therapy clinical trial at UNC Chapel Hill. Our fundraising efforts continue as we work to provide grants towards additional research for MPS II. As the number of current MPS II clinical trials increase, we are exploring how we can help enable the greatest number of patients access to these treatments. Our efforts towards research will not end until a cure has been found.
Our Mission and Vision
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.
Stay tuned to our social channels for the latest developments.