Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Transthyretin amyloidosis is a rare, progressive disease characterized by the abnormal buildup of amyloid deposits composed of misfolded transthyretin protein in the body's organs and tissues; this build up is called amyloidosis.

ATTR amyloidosis can impact numerous organs and tissues in the body, including the peripheral nervous system, and organs such as the heart, kidneys, gastrointestinal tract, and eyes. ATTR-CM is a presentation of the disease that affects the heart and leads to restrictive cardiomyopathy and progressive heart failure.

ATTR-CM occurs when transthyretin, a transport protein that naturally circulates in the blood, becomes unstable, and dissociates into monomers, which misfold. The misfolded protein aggregates into amyloid fibrils which build up in the heart and causes the heart muscle to become stiff, eventually resulting in heart failure.