rare diseases
Seldom-seen ailments
Rare diseases are medical conditions affecting a small percentage of the population—in the US, fewer than 200,000 people; in Europe, fewer than 1 in 2,000.
Despite individual rarity, thousands of rare diseases exist collectively, affecting millions worldwide.
Many are genetic, chronic, and difficult to diagnose.
The orphan drug program incentivizes treatments for rare diseases.
- Behçet's diseaseChronic multi-organ vasculitis
- genetic disordersConditions caused by mutations
- undiagnosedNot yet medically named
- medical mysteryUnexplained illness or condition
- metabolic disordersImpaired biochemical processes in cells
Tay-Sachs disease · hereditary conditions · misdiagnosis · Niemann-Pick disease · porphyria · fragile X syndrome · genetic counseling · gene therapy · genetic · incurable · one in a million · cystic fibrosis · debilitating · hope · patient advocacy · support groups · awareness ribbon · chronic
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