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Breakthrough 科学
We relentlessly pursue bold science to translate genetic discoveries into new medicines that advance the future of human health.
We relentlessly pursue bold science to translate genetic discoveries into new medicines that advance the future of human health.
We listen to and learn from patient communities to collaborate on solutions with the potential to help improve care.
Our talented and dedicated people represent the best the biotech industry has to offer and have helped shape a culture focused on working together to deliver for the patients we serve.
Since our founding in 1997, we’ve applied our scientific expertise in understanding the underlying causes of genetic conditions to create transformative medicines, using a number of treatment modalities. Our approved medicines treat achondroplasia, severe hemophilia A and several rare inherited and lysosomal storage diseases. We’re also advancing an industry-leading pipeline of investigational medicines that are in various stages of clinical and preclinical development.
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