Tag: Transthyretin

In May 2019, the FDA approved Tafamidis, marking the first time a medication was specifically approved to treat transthyretin amyloid cardiomyopathy (ATTR-CM). This article explores the specific clinical scenarios for which this groundbreaking drug is prescribed, detailing the disease it targets and its significant impact on patient outcomes.

In a significant development for patients with a specific type of amyloidosis, recent FDA approvals have provided new and transformative options for treatment. This includes new drugs to treat amyloidosis, specifically targeting transthyretin-mediated cardiac amyloidosis (ATTR-CM) with different mechanisms of action.

First approved in 2022 for hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy, **vutrisiran** (brand name Amvuttra) is a gene-silencing medication that offers a new approach to treatment. It works by targeting the root genetic cause of the disease, preventing the production of the toxic protein that causes nerve and heart damage.