Investigational Therapy

Sometimes called "compassionate use", expanded access is a potential pathway for a patient with a serious or life-threatening disease or condition to try an…

Gastrointestinal (GI) perforation is remarkably rare in patients with light chain (AL) amyloidosis and has not yet been reported in patients with AL amyloidosis…

Systemic amyloidosis occurs when one of a growing list of circulating proteins acquires an abnormal fold, aggregates and gives rise to extracellular amyloid…

Cardiac amyloidosis (CA) has been believed to be a rare disease for a long time, but recent sophisticated diagnostic modalities demonstrate that a considerable…

Transthyretin (TTR) amyloidosis (ATTR amyloidosis) is a multisystemic, multigenotypic disease resulting from deposition of insoluble ATTR amyloid fibrils in various…

Amyloid cardiomyopathy should be suspected in any patient who presents with heart failure and preserved ejection fraction. In patients with echocardiographic…

Amyloidosis is a rare condition that used to almost always be fatal, but doctors are finally making progress in treating it. "I had so many symptoms, and I really…

Amyloidosis is a disorder in which certain proteins abnormally change their shape in a process called "misfolding." The misfolded proteins accumulate together and…