That’s why we partner with a range of international and regional rare disease advocacy groups to learn and co-develop programs to empower patients and caregivers. Find resources, advocates, and allies to help you on your journey.
We encourage patient organizations to visit our sponsorship portal for more information. Our goal is to provide compliant support to organizations and connect them with resources for the benefit of the rare disease community.
As the science evolves, our focus is on finding therapies for the following key rare disease areas:
Inborn Errors of Metabolism
Sickle Cell Disease
Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)