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National Organization for Rare Disorders

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Biotech

Small pools, big ideas: Rare disease community reimagines trials

To get rare disease drugs through FDA scrutiny and into the hands of patients, researchers and biopharma companies are getting creative.
Angus Liu Apr 17, 2026 3:00am
singer Celine Dion performing on the Eiffel Tower during the opening ceremony of the Paris 2024 Olympic Games

In the wake of Céline Dion's comeback, SPS marches toward a cure

Aug 15, 2024 2:45pm