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.

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CRYSVITA® (burosumab-twza) logo

Patients and healthcare providers, learn more at

Report a side effect

You may report side effects to the FDA at 1-800-FDA-1088 or

You may also report side effects to Kyowa Kirin at 1-844-768-3544.


MEPSEVII® (vestronidase alfa-vjbk) logo

Patients and healthcare providers, learn more at

Read the U.S. Prescribing Information for MEPSEVII

UltraCare: Our program to help facilitate patient access to our medicines. Learn more at

Discover more about Ultragenyx:

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Latest news

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Pipeline Med Card


Discover our portfolio of therapies in development.

View our pipeline