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- - - To transform the lives of as many people living with rare diseases as possible, we dedicate ourselves every day to the development and delivery of treatments of rare diseases where none currently exist.
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- - - We develop therapies for rare and ultrarare genetic diseases by exploring an array of drug modalities to select the best treatment strategy for each genetic disease we are studying.
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- - - We have built a talented team and a vibrant, people-first culture. We share a passion for helping those living with rare diseases.
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May 17, 2024

Hopes, Fears for Orphan Drug Act

Ultra-Committed