Program 02 · The data infrastructure
Patient Registry
Family-controlled, IRB-approved records and natural-history data, federated so many small cohorts become one research surface.
What it does
- 01
Collect natural-history data that a trial actually needs: longitudinal audiograms, imaging, and hearing trajectory over time.
- 02
Keep records family-controlled and IRB-approved. Families own their data and consent per use. We will never sell it, because it is not ours to sell.
- 03
Federate on the RARE-X model so a small STRC cohort can sit inside a much larger, shared research surface, and extend to adjacent hearing-genetics conditions later.
Why it matters
A single rare-disease registry is too small to power a study. Federated, many become large enough to matter. Natural-history data is the raw material regulators now accept as an external control arm, which is how tiny populations run rigorous trials. RARE-X already federates data across 80-plus advocacy groups and 57 disease communities.
Where it stands
Planning. Year-one targets are a RARE-X agreement, the first families enrolled, and independent IRB approval. Patient data never lives in the public vault. Aggregates only.