Disease Focus

Transthyretin amyloidosis is a rare, rapidly progressive disease caused by a build-up of misfolded transthyretin proteins produced by the liver.

Hereditary angioedema is a rare, hereditary condition associated with frequent episodes or attacks of painful swelling in various parts of the body.

Alpha-1 antitrypsin (AAT) deficiency is a genetic disease in which the body does not make enough AAT, a protein that protects the lungs and liver.