Why We Show Up

Living with a rare disease is difficult, and while it is rare, the difficulties are not rare to us. Our team has a deep appreciation for the patients who have previously participated or are currently enrolled in our clinical trials. We recognize the valuable time and effort committed to our research efforts—contributions that are essential to finding novel solutions for diseases, such as NPC and Gaucher disease.

We are stronger together

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

In addition to the relationships we have with the patients we serve, we also value the relationships we have with community leaders and non-profit organizations that are working to improve the lives of people affected by neurodegenerative rare diseases.