Breakthrough Science

We relentlessly pursue bold science to translate genetic discoveries into new medicines that advance the future of human health.

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Focus on Patients

We listen to and learn from patient communities to collaborate on solutions with the potential to help improve care.

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People-First Culture

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.

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澳门正规博彩十大排行平台 is a global biotechnology company with eight marketed products that treat life-threatening conditions.

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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Join Our Team

Come work with us. Search our global career opportunities to learn how you can make your mark.

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