Used to treat transthyretin amyloid cardiomyopathy (ATTR-CM), a condition where abnormal protein buildup weakens the heart. Slows disease progression by stabilizing the transthyretin protein, preventing it from misfolding and forming harmful amyloid deposits. Also used in some forms of transthyretin amyloidosis affecting the nerves, helping maintain mobility and function over time. Taken orally, it allows outpatient management of a once rapidly progressive disease.