Why join the cystinosis program?
Reduce the time for cystinosis 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 cystinosis research
Cystinosis is a rare genetic disorder that causes the buildup of an amino acid called cystine in organs, including the kidneys and brain. There are three different forms known as nephropathic cystinosis, intermediate cystinosis and non-nephropathic (or ocular) cystinosis.
Nephropathic cystinosis develops when children are infants and is by far the most common and severe form. Through treatment and improvements in kidney transplantation, this type of cystinosis has shifted from a fatal kidney disorder to a chronic, multisystem disorder.
The latest on our research: We are jumpstarting our cystinosis program to accelerate research on the disorder to ultimately improve treatment options. We welcome all parents or patients interested in participating and spreading the word to the cystinosis community.
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.