Small interfering RNAs (siRNAs) are used to treat conditions like hereditary transthyretin-mediated amyloidosis (hATTR), primary hyperoxaluria type 1 (PH1), and high cholesterol. They also have other uses, such as managing acute hepatic porphyria (AHP). siRNAs work by targeting and degrading specific RNA molecules, which prevents the production of disease-causing proteins. These medications are typically available as injections. Examples include Onpattro (patisiran), Givlaari (givosiran), and Leqvio (inclisiran).
