We are dedicated to helping those affected by Niemann-Pick disease type C (NPC): patients, parents, carers, physicians, and communities.
We were purpose-built for patients and recognize the urgency felt by the community. We partner with 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 NPC.
We will continue to engage actively with the passionate community of patients, caregivers, clinicians, and regulators over the coming months. We care deeply about supporting patients and are committed to improving the lives of people with NPC.
Moving forward together
We are dedicated to supporting the Expanded Access Program and future research and development of adrabetadex for patients living with NPC.
#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 as part of the Expanded Access Program, when appropriate. We are also working to find a path to advance the development of adrabetadex.
We continue to support the Expanded Access Program and continue to work with our regulatory and clinical partners to supply adrabetadex for EAP use.
#BuiltForPatients
Advancing research
We recognize the NPC community will leave no stone left unturned in efforts to uncover the best possible treatment options. 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 apprised with the latest developments.