Tag: Amyloidosis

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.

An estimated 4,000 people develop primary amyloidosis in the U.S. each year, a rare disease that has seen a recent surge in treatment options [1.10.3]. So, what is the new drug for amyloidosis? The answer depends on the type, with several groundbreaking therapies approved in late 2023 and 2024 [1.3.1, 1.4.1].

Hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) is a rare and progressive genetic disorder, estimated to affect between 10,000 and 40,000 people globally. This condition is caused by a gene mutation that results in abnormal protein deposits that damage the nervous system and other vital organs.

In transthyretin-mediated amyloidosis (ATTR), a progressive and often fatal disease, the transthyretin (TTR) protein misfolds and forms harmful amyloid deposits. The therapeutic effectiveness of Vyndaqel hinges on its unique ability to disrupt this process, making understanding what is the mechanism of action of Vyndaqel crucial for both patients and clinicians.

In November 2024, the U.S. Food and Drug Administration (FDA) approved Attruby (acoramidis) to treat adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). This oral medication serves a crucial role in reducing death and hospitalizations related to heart problems caused by this rare and serious condition. If you're asking, **'What is Attruby for?'**, this guide will explain its purpose, function, and other important information for patients.