100 Patient Project: Unlocking MPS
We have an exciting new project that will shed light on MPS II – making the path to finding a treatment faster. We need you! Every journey is a piece of the puzzle.
Click here and register on behalf of your child, fill out two surveys, and connect your child’s electronic health records.
After we receive your child’s questionnaires and EHR we will see if they qualify for whole genome sequencing and we will offer that to you for free. Need help? Contact [email protected].
Dear Members of The MPS II (Hunter Syndrome) Community:
We are excited to announce a groundbreaking research partnership we believe holds great promise to improve the lives and disease outcomes of the MPS and ML community. This partnership includes:
- National MPS Society, Inc.
- Genetic Alliance, a 501(c)(3) genetic disease and research charity
- Luna, a platform that connects families, registries/communities/patient advocates, and researchers to advance health discovery
- Takeda Pharmaceutical Company
The 100 Patient Project is an inaugural part of a new registry for MPS and ML: Unlocking MPS. With an initial focus on MPS II, we are launching the 100 Patient Project to harness the revolutionary insights that can come from Whole Genome Sequencing (WGS) to better understand how genetic variation can inform future improvements to MPS disease management. Sign up to join us today!
We invite those with MPS II or their caregivers to join the Luna portal created for the 100 Patient Project and the Unlock MPS Registry. You will fill out two introductory surveys, one with very basic information and a second Registry Intake Survey with details of your journey with MPS or ML. 100 individuals with Hunter syndrome (MPS II) will be selected to provide a blood sample from which a WGS will be derived. Individuals should have their electronic health records linked in order to be considered for WGS.
The medical record data and the DNA will be housed in the most secure manner possible for medical information. The genetic data, in particular, will be held according to the most advanced standards, including those of the State of California and the European Union. Luna takes data security seriously. The safety and use of its members’ data are critical. The research data remains within the electronic platform and cannot be downloaded separately. Additionally, before researchers see any data, it is completely anonymized and cannot be used to identify a patient.
Why yet another registry?
By newly focusing on a subset of individuals with MPS II, we hope to acquire a more thorough dataset that will unlock MPS II research and discoveries to help our patient community. A small dataset of 100 patients that includes both complete medical records and a WGS, will be complete in ways that other natural history and clinical databases are not. This new level of completeness will increase the chances of discovering important new information.
What control do I have of my information after it is deposited with Luna?
You control ultimate access to the data. By participating, you join as a patient partner through Luna, an innovative Public Benefit Corporation organization. As a registry member, you control ownership of your data and access to that data. Patients have the right to not only withdraw from a research project, but can even delete their accounts and have their data destroyed if they wish.
How is access controlled?
To ensure the approved certification, legitimacy, ethics, and security of proposed research protocols, all research access to the data will be overseen by the National MPS Society, .
What are the benefits of participating in the 100 Patient Project?
There are benefits to those taking part and the wider community; some tangible, others intangible.
- You will have the satisfaction of taking part in a patient-directed research registry
- You will be able to see any of the biomedical discoveries from this research
- You may potentially benefit from better treatment and management of MPS and ML
- If you are selected to take part in the inaugural 100 Patient WGS:
- You will have access to your entire genome sequence
- You will also receive a voucher for an online counseling session with a licensed genetic counselor
Where do we go from here?
The Unlocking MPS Registry is managed as part of a research protocol headed by the National MPS Society’s Dr. Matthew Ellinwood, Chief Scientific Officer and Terri L. Klein, President/CEO as a Co-Investigator. Project funding is supplied by Takeda through independent grants to the Genetic Alliance and Luna. The actual research (as opposed to just collecting survey data, records, and WGS), will also be headed up by Dr. Ellinwood, with this and future research access overseen by the National MPS Society.
We hope you are excited about this new project as we are and that you start the process of signing up for a Luna registry today to register and complete our intake surveys.
With Hope for Brighter Days for All of Our MPS and ML Community!
Matthew Ellinwood and Terri Klein
To access the intake survey and begin enrollment into this critical project, click “Join Us” below.
If you have difficulty accessing the link on your browser, please visit this direct link to begin: