Child giving high-five

Partnering with patients throughout their health journeys

Partner advocacy organizations worldwide
Industry-leading support programs for U.S. patients
Global online educational resources

Our commitment to patients extends beyond developing therapies. We also provide support and advocacy programs, educational materials, and other resources to inform and empower patients and their families.

Image of Pompe disease patient

Commitment to patients

Our work extends beyond developing therapies. We’re committed to helping patients obtain access to our treatments by offering a variety of support programs to eligible patients.
Image of Pompe disease patient and her mother

Patient advocacy

We work with organizations around the world who share our mission to advocate for the needs of patients.
Gaucher disease patient family

Lysosomal storage disorder registries

These large, often multinational databases of clinical data, aggregate knowledge to help improve understanding of rare diseases that would be otherwise difficult to study.
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Reporting side effects

Gaucher patient

Patient access to treatment

Image of Thyroid cancer patient

Educational websites

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