Zevra has sold its US Food and Drug Administration (FDA) priority review voucher (PRV) for $150m to an undisclosed party. The ...

Zevra used the awareness day to roll out the “Learn NPC, Read Between the Signs” disease awareness campaign. The biotech won ...

Several rare disease patient populations received their first-ever FDA-approved drug since Rare Disease Day last year, ...

ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, ...

The brain drug developer is continuing a trial despite a recommendation it be stopped for futility. Elsewhere, Eli Lilly ...