Why join the Sanfilippo syndrome program?
Reduce the time for Sanfilippo Syndrome - MPS III drug development
All your medical data in one place
Follow new clinical trials
Make your de-identified health information part of multiple research projects
Our work with Sanfilippo syndrome research
Sanfilippo syndrome, also known as mucopolysaccharidosis (MPS) type III, is a severe lysosomal storage condition that occurs when patients cannot break down long chains of sugar molecules. This causes devastating damage to the central nervous system. There are four subtypes of Sanfilippo syndrome (types A, B, C, and D). Early in childhood, patients experience severe developmental delay and regression. Over time, patients decline and many Sanfilippo patients do not survive past their teenage years. There is currently no cure for Sanfilippo syndrome.
The latest on our research: We are kicking off work with the Cure Sanfilippo Foundation on a new study. We want to make this study as strong as possible to accelerate more research, so we are currently recruiting more Sanfilippo families to join in this effort. We will share more details as the research progresses.
How it works
1. Sign up
Create your private account and review our research consent (takes about 10 minutes)
2. We get to work
We do all the behind-the-scenes work to retrieve and process your records
3. Empower research
Track your contribution to research projects and, based on your records, find out if you qualify for new clinical trials based on your records
Our values & principles.
We believe that the only way to keep data accessible to the researchers who need it is by enabling patients to own and control their own data. Patients decide for themselves whether they want to privately and securely contribute to research, rather than having the decision made on their behalf.
We aim to make useful data accessible to all researchers who have legitimate research questions or research programs. For academic research, we offer access without charge. We take research ethics seriously, and we have policies in place to ensure that research is conducted with integrity.
We don’t like to take sides—we give all members of the community the option to get involved. Success in rare disease research takes a village and we always welcome additional collaborators who can advance and benefit from the mission.
Our primary mission is to accelerate drug development, so we prioritize generating insights specifically relevant to therapeutic research. We invest deeply in quality, security, and compliance to make this a reality.