SMALL INTERFERING RNA (SIRNA) BASED THERAPY

Course overview

PATISIRAN, GIVOSIRAN, LUMASIRAN, AND INCLISIRAN ARE FDA-APPROVED FOR MANAGING RARE METABOLIC AILMENTS. ALL FOUR AGENTS ARE SMALL INTERFERING RNA (SIRNA) BASED THERAPIES THAT EXERT THEIR EFFECTS BY RNA INTERFERENCE (RNAI) OF THEIR TARGET MRNA. THIS ACTIVITY WILL HIGHLIGHT THE MECHANISM OF ACTION, ADVERSE EVENT PROFILE, AND OTHER KEY FACTORS PERTINENT TO INTERPROFESSIONAL TEAM MEMBERS IN MANAGING ADULT PATIENTS WITH HEREDITARY TRANSTHYRETIN AMYLOIDOSIS (HATTR), ACUTE HEPATIC PORPHYRIA (AHP), PRIMARY HYPEROXALURIA TYPE 1 (PH1), AND LOWERING OF LOW-DENSITY LIPOPROTEIN CHOLESTEROL (LDL-C) IN SUBJECTS WITH HETEROZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA (HEFH) OR CLINICAL ATHEROSCLEROTIC CARDIOVASCULAR DISEASE (ASCVD).