Our commitment to patients is an active promise to join their fight—to pursue unmet needs and to work quickly and creatively to deliver meaningful results.
We put patients first in everything we do, whether it’s the way we develop our medicines, design our packaging, or help them adjust to a new medication regimen. Living with a rare disease is not easy. We hope to provide the important information and support so patients don’t walk this journey alone.
Disease areas
We address serious and rare diseases that are traditionally overlooked and underserved.



Patient resources
Connect with others walking the same journey and access practical information for support and assistance.
Commitment to access
We’re dedicated to providing patients with access to our medicines, and we work with numerous stakeholders to do so.