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.


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

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Discover our portfolio of therapies in development.

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