Our Mission & Vision

Bringing life-changing therapeutics to people living with rare diseases

Our Vison

To become a leading rare disease therapeutics company that is driven by patient insights and innovation to make a transformational impact on the people we serve.

Our Mission

We redefine what is possible in bringing life-changing therapies to people living with rare diseases.

Our Values

Children living with Niemann-Pick disease type C

Patient Centricity
prioritizing patient needs

Accountability
delivering on commitments consistently

Integrity
prioritizialways doing what is right

Innovation
transforming possibilities into reality

Courage
acting with strength and conviction