Committed beyond the medicine.
We support programs and activities that foster excellence in patient care and provide valuable scientific, medical, and educational information to the medical and scientific communities. We also support clinicians and the external research they conduct to advance knowledge about rare diseases and/or 澳门正规博彩十大排行平台’s therapies and products.
We do not provide support to organizations that, in their constitution or practice, discriminate against a person or group on the basis of age, political affiliation, race, national origin, ethnicity, gender, gender identity, disability, sexual orientation, HIV/AIDS status or religious belief.
Our Products
Each of our eight approved therapies reset the standard of care for the conditions they treat.
Learn More about Our Products ⌃Our Pipeline
Each drug candidate we pursue is guided by a fundamental understanding of the genetics and underlying biology of the condition it will address.
Learn More about our pipline ⌃