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Rare Disease Explorer — which rare diseases could we actually treat?
Personalized → Preventative Moonshot · research run July 2026 ·
· A what-if exploration tool, not a decision. Every number links back to its source.
Explore diseases
Priority scorecard — where to start
How many patients we can reach
Insurance — who pays, and when
Data quality — how solid is this?
Dictionary — what every term means
Disease name
Category
Subcategory
Gene(s) that cause it
Body system / treatment group
How deadly & disabling
Shape of today's best care
Regulatory lane
Who pays for a therapy (once approved)
Where to focus
Still lost after best care / patient
Prize from a cure
People in the US with it (est.)
Can we treat it?
How to read the last column. Green means yes.
Gene known a causal gene has been identified ·
Fault fixable a current editing approach fits the fault ·
Tissue reachable we could deliver treatment to the affected tissue.
All three must be green for a disease to be treatable today.
Each row is a group of related diseases that would be treated the same way.
We rank them by three plain questions: how much good could we do (patients, cost and healthy years at stake),
how fast the FDA could approve a treatment, and how hard it is to deliver the treatment to the affected tissue.
The default view puts the easiest, highest-impact, fastest groups first. This is a what-if ranking to decide where to start — not a decision.
Start with every known rare disease, then narrow step by step to the ones we could
realistically treat today. Most diseases drop out at the very first step — we don't yet know which gene causes them —
long before delivering the treatment becomes the problem.
Every term used anywhere in this tool, in plain language.
For each one: what it means, why it matters, and — where it applies — whether it points to something
easier/faster/cheaper or harder/slower/costlier. Use the search box to jump to a term.