Why join the Niemann-Pick program?
Reduce the time for Niemann-Pick disease type C 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 Niemann-Pick research
Niemann-Pick disease type C (NPC) is a rare, genetic disease that affects the body’s ability to metabolize fat within cells. It is in a group of disorders called lysosomal storage diseases. In NPC, cholesterol and other fats build up and affect the liver, spleen, lungs or brain.
Niemann-Pick can occur at any age but primarily affects children. The disease has no known cure, and most children with NPC do not live to adulthood.
The latest on our research: We are currently recruiting families to help make this NPC research database as strong as possible, in partnership with Firefly Fund. We hope to fuel multiple future research projects and studies, and will keep participants updated on our progress.
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.