Developing innovative therapies for those living with serious rare and ultrarare genetic diseases
Our approach has led to the delivery of multiple first-ever rare disease treatments at a faster speed than the industry average.
CRYSVITA® (burosumab-twza)
Patients and healthcare providers learn more at CRYSVITA.com
Read the U.S. Prescribing Information for CRYSVITA
UltraCare: Our program to help facilitate patient access to our medicines. Learn more at UltraCareSupport.com
DOJOLVI® (triheptanoin)
Patients and healthcare providers learn more at DOJOLVI.com
Read the U.S. Prescribing Information for DOJOLVI
UltraCare: Our program to help facilitate patient access to our medicines. Learn more at UltraCareSupport.com
MEPSEVII® (vestronidase alfa-vjbk)
Patients and healthcare providers learn more at MEPSEVII.com
Read the U.S. Prescribing Information for MEPSEVII
UltraCare: Our program to help facilitate patient access to our medicines. Learn more at UltraCareSupport.com
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Latest News
Get the latest information related to our company, our pipeline, and our medicines.
Visit the newsroom
You may also be interested in:
Latest News
Get the latest information related to our company, our pipeline, and our medicines.
Visit the newsroom