Putting patients first

Empathy for patients drives our urgency.

Riley, in memoriam, lived with PKAN Syndrome (Pantothenate Kinase-Associated Neurodegeneration)

Our Approach

Patient advocacy is a partnership.

Collaboration starts with conversation. We connect with relevant organizations early to share our development plans, exchange ideas, and ask for feedback.


Community members aren’t just patients or customers—they’re our partners. Listening to them, their families, and their caregivers helps shape our treatment priorities and gives us a richer picture than clinical data alone. Where research meets real experience, our work is positioned to make deeper, more meaningful impact.

Our Promise

We work to build honest, genuine relationships.

We know we’re often meeting people at deeply vulnerable moments. So we don’t mince words or spout false hope. We show up consistently and speak transparently about what we know and what could be done better.


By striving to lead with integrity, we uphold the independence of each organization we work with. We measure our success not just by the medicines we develop, but by the trust we earn with the people we’ve developed them for.

Patient Stories

Alexis & Jackson, living with ADH1

Conditions

Get to know the conditions we’re focused on.

Looking for Support?

Connect with advocacy organizations.