Alexander Disease


A Study to Evaluate the Safety and Efficacy of ION373 in Patients With Alexander Disease (AxD)

Severe Hypertriglyceridemia

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A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)

Phlebotomy Dependent Polycythemia Vera

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A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera

Amyotrophic Lateral Sclerosis

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Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)

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Therapeutic Conditions


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.