Our Clinical Studies
Receiving a devastating diagnosis can be overwhelming, especially when there are no approved therapies available and limited information to be found. It is important to know that you are not alone.
Alexander Disease
A Study to Evaluate the Safety and Efficacy of ION373 in Patients With Alexander Disease (AxD)
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Therapeutic Conditions
Neurological
Hematology
Other
Working with our Community
Through our collaborations with patient advocacy organizations and communities worldwide we work to identify and understand the most urgent needs of people living with severe diseases and their caregivers. As experts in living with their disease, the patient and caregiver perspectives inform our approach to research.
