Learning from innovation in rare disease science to create a more equitable health future
In the last 30 years, researchers at a leading company have transformed a largely unaccepted science into first-of-their-kind therapies. Its medicines have brought hope to rare disease patients who had few to no options — but the work is far from over. Read about this company's novel approach to R&D and how it’s harnessing these learnings to address the unmet needs and equity challenges within the rare disease community.
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Reviewed by Knowledge World
on
February 25, 2022
Rating: 5
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