vutrisiran (Pending FDA Approval)

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Dosing & Uses

Transthyretin-Related Amyloidosis

Pending FDA approval for treatment of polyneuropathy caused by hereditary transthyretin-related amyloidosis (hATTR)

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Pharmacology

Mechanism of Action

Subcutaneously administered, RNAi therapeutic targeting transthyretin (TTR); it is designed to target and silence specific messenger RNA, blocking the production of wild-type and variant TTR protein before it is made

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Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.