Fajansa, Pompe disease, India

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.
Image of atopic dermatitis patient
Reporting side effects
Gaucher patient
Patient access to treatment
Image of Thyroid cancer patient
Educational websites

This website uses cookies to track its audience and improve its content. By continuing to browse this website, you agree to the use of such cookies.

Click here for more information on cookies.