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.
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.
NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of AKCEA-TTR-LRx in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of AKCEA-TTR-LRx in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
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)
A Study of AKCEA-APOCIII-LRx Administered to Patients With Familial Chylomicronemia Syndrome (FCS)
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A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Participants With Chronic Heart Failure With Reduced Ejection Fraction
A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)
Please notify me when the study is enrollingThrough 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.