FDA-Approved

Which FDA-approved rare disease treatment is identifying patients fastest?

Featured Race
Cystic Fibrosis vs Fabry Disease: How Do Their ClinVar Discovery Rates Compare?

Two blockbuster rare disease franchises. One has 74× higher prevalence. When you normalize ClinVar submissions for population size, the contrast is striking.

CFTR (Vertex) $10.9B revenue
vs
GLA (Sanofi) €1.0B revenue
View comparison →

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