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'Rare' Disease? In the U.S., a rare disease is one that affects fewer than 200,000 people. But with around 10,000 rare diseases, that number adds up quickly.

The survey primarily focused on rare disease patients between the ages of 16 and 23, as well as caregivers between 25 to 55 years old. Hemophilia, albinism, cystic fibrosis and Gaucher disease were ...

Europe risks ceding its leadership in rare disease innovation unless it urgently recalibrates its incentive framework, warns Enrico Piccinini, Senior Vice President EU & International at Chiesi.

The Senate Parliamentarian reversed course on a small provision in the One Big Beautiful Bill Act that could boost cures for rare diseases.

A rare disease is classified as one that affects fewer than 200,000 people. But the sum of them looks very different: 7,000 rare diseases affect more than 30 million Americans.

Scientists have corrected gene mutations in mice causing an ultra-rare disease by editing DNA directly in the brain with a ...