Abstract
Disease-modifying therapies such as transthyretin (TTR) gene-silencing drugs and TTR tetramer stabilizers have been developed for hereditary transthyretin (ATTR) amyloidosis, a major type of autosomal dominant hereditary amyloidosis. Recently, a second-generation TTR gene-silencing drug, vutrisiran, was approved for treating patients with hereditary ATTR amyloidosis in Japan. This new drug significantly reduced the patient's physical burden.
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have