For families
If you just found out, you are not as alone as you feel.
I am Misha's father. When we got his diagnosis, we had never heard of the gene either, and there was no one to call. This page is for any family with a rare genetic disease. It is the thing I wish someone had handed us.
You have never heard of this gene. Neither had we.
The hardest part at the start is not the test result. It is the feeling that you are the only family in the world with this. That feeling is real, and it is also wrong. Whatever the gene, there are more of us than the silence suggests, and finding each other is the first thing that helps.
What is actually true today
Not the worst thing you read at 2am. This.
Most rare disease is tractable
About 72% of rare diseases are genetic, usually a single gene. That is the kind of problem computation and gene therapy are built for, not a lost cause.
Your data stays yours
The registry is family-controlled and IRB-approved. You own your records and consent to each use. We will never sell your data, because it is not ours to sell.
Someone is working on it
That is the whole reason this foundation exists. You are not waiting alone for a system that forgot you.
Honest hope, not a promise
Real, early science keeps landing in rare disease. For our own gene, STRC, a 2026 gene therapy restored hearing to near-normal levels in a mouse model. That is not a cure, and I will not pretend it is. It is proof that moving now beats waiting, and the same is true for the gene your family carries.
The most useful thing you can do
Families who take a concrete action feel less powerless, and the action genuinely moves a future treatment closer. Here is yours.
Add your child to the registry
It takes about ten minutes, and it is the single most useful thing a family can do to make a future trial possible.
Find the others
Peer-parent introductions, a community call, and newly-diagnosed guidance written for parents, not clinicians.
Tell us what matters
Family input decides which research gets funded first. You know what a good day looks like better than any committee.
One family per disease. That is how this scales.
STRC is our first branch because it is the case we know from the inside. The pipeline, the registry, and the community were built to be reused, and every new disease starts the same way ours did: with one family who refuses to wait.
We are recruiting family ambassadors
An ambassador is a parent who leads the branch for their own child's disease: the newly-diagnosed guide, the family network, and the call on what gets researched first. You bring the lived expertise. We bring the pipeline, the registry infrastructure, and the fundraising model. That is how one rare disease becomes many.
Start a branch for your diseaseWrite to me
Whatever gene your child carries, even one no one has named yet, I want to know you exist. The registry and the community start with you.