Our Philosophy:
We are Stronger Together

We are pioneering a new kind of treatment approach harnessing the potential inside human cells. As we advance our clinical development programs, we look to those who are living with the diseases we aim to treat for inspiration and guidance.

Please read our statement to the NPC Community from June 2021 regarding the FDA review of our new drug application for arimoclomol in NPC.

Regan Sherman leads our engagement with patient communities around the world.

As a former advocate herself, Regan seeks to understand and elevate the needs of people living with orphan diseases to guide our efforts throughout the drug development process, from clinical trial design through to access.

"Our team is grounded in the belief that people living with rare and orphan conditions and their families are disease experts and key stakeholders in the development of new therapies. We are grateful for more than 10 years of collaboration with the Niemann-Pick community that has helped bring us to our first regulatory submission. Support and guidance from patient communities in all of the disease areas we're involved with are critical to our continued success."


We value the relationships we have with community leaders and non-profit organizations that are working to improve the lives of people affected by neurodegenerative orphan diseases. Click on the links below to learn more about organizations taking the lead in supporting and advocating for these communities.

Neuromuscular diseases:

Lysosomal storage diseases: