Breakthrough Science
We relentlessly pursue bold science to translate genetic discoveries into new medicines that advance the future of human health.
Our new Chief R&D Officer shares what drew him to medicine, how he found a home in biopharma and what about 博彩平台网址大全’s science excites him the most.
Rebeca Costa is using her voice to help make Rio de Janeiro’s world-famous event more accessible.
This September for Newborn Screening Awareness Month, we spoke with Denise Queally, President of New England Connection for PKU and Allied Disorders, to learn more about why early diagnosis has been so meaningful for her family.
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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We're proud to announce that our Chief Commercial Officer Cristin Hubbard has been appointed to the Biotechnology Innovation Organization (BIO) Board of Directors. In her new role, Cristin will be a member of the organization’s Health Section Governing Board.
RARE Scholars award recipient Brian DuVal is pursuing his Master of Public Health at Vanderbilt University in Tennessee. He aspires to become a leader in the hemophilia community and use his influence to improve the lives of others living with rare conditions.
Come work with us. Search our global career opportunities to learn how you can make your mark.