PATIENTS AND FAMILIES
We put the patient first in all that we do.
We understand that having a rare disease brings unique challenges but know that you are not alone.
Every day we work to advance safe and effective therapies with the potential to change the course of the disease for our patient communities.
Our work goes beyond delivering innovative solutions. We collaborate with patients, families, advocacy groups and physicians in order to:
- improve disease education and awareness
- to expand testing and diagnosis
- ease the burden that can come with living with a rare metabolic disease
- connect families with others to provide support and build community