We are dedicated to helping everyone affected by Niemann-Pick Type C1 (NPC1): patients, parents, carers, physicians, and communities.
We were purpose-built for patients and recognize the urgency felt by the community. We partner with all our stakeholders to more deeply understand what’s most important and how we can build a path forward.
We are here for you
It is our goal to move adrabetadex* forward in its development in the most efficient manner possible and in consideration of all those suffering with NPC1.
We look forward to engaging with this passionate community of patients, caregivers, clinicians, and regulators over the coming months. We care deeply about supporting access for patients and are committed to supporting efforts to improve the lives of people with NPC1.
Fighting on your behalf
We are dedicated to supporting the continuation of the Expanded Access Program and future research and development of adrabetadex for patients living with NPC1.
#BuiltForPatients
Working with our partners
Our connection to each facet of the community is of the utmost importance to us. We believe that collaboration will enable us to progress our understanding of the disease and potentially provide new diagnostic and treatment options for patients and their families.
#NoStoneLeftUnturned
Expanded Access Program
We are #BuiltForPatients which means we are committed to providing access to adrabetadex outside of participation in a clinical trial, when appropriate, at the same time as we’re working to find a path to advance the development of adrabetadex.
To enable the continuation of the existing Expanded Access Program, we have secured additional drug supply and will be working with our regulatory and clinical partners over the coming weeks and months.
#BuiltForPatients
Advancing research
We recognize the NPC community will leave no stone left unturned until a treatment is found. We are conducting a full evaluation of the adrabetadex clinical data available to us and will provide the community updates as we work through this process.
#NoStoneLeftUnturned #TheRightThingToDo
Collaboration
Our initiatives are set up to consider the spectrum of challenges associated with this disease. We recognize also that collaboration is critical to addressing those challenges.
#TheRightThingToDo
Join our mailing list
We will keep you to do date with the latest developments.