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.
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of AKCEA-TTR-LRx in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of AKCEA-TTR-LRx in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
Macular Degeneration
GOLDEN STUDY: A Study to Assess Safety and Efficacy of Multiple Doses of IONIS-FB-LRx in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration (AMD)
Familial Chylomicronemia Syndrome
A Study of AKCEA-APOCIII-LRx Administered to Patients With Familial Chylomicronemia Syndrome (FCS)
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Therapeutic Conditions
Cardio-Renal
Metabolic
Neurological
Cancer
Ophthalmology
Pulmonology and Allergy
Hematology
Other
Upcoming Clinical Studies
Steatohepatitis, Nonalcoholic
A Study to Assess the Safety, Efficacy, and Pharmacokinetics of Multiple Doses of ION224
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.
