Ionis Pharmaceuticals

Serum amyloid P component (SAP) is a normal protein found in the blood that binds to amyloid deposits. In healthy people there are very small amounts of SAP and…

In AL amyloidosis, an abnormal protein called amyloid is produced and accumulates in tissues and organs. The build-up of amyloid protein is called an amyloid…

Steroids are drugs which mimic certain hormones in the body that help to regulate and control the way the body develops and functions. There are several types of…

Fatigue is a feeling of near constant exhaustion that differs from the everyday tiredness that comes with the demands of a busy daily life. Unlike ordinary…

Alexion Pharmaceuticals (ALXN) has been one of the greatest growth stories in the ultra-rare disease space over the past decade, with their blockbuster C5 inhibitor…

Amyloidosis is a disorder in which misfolded native proteins deposit extracellularly and lead to organ damage. Although the various types of amyloidoses share in…

The term 'amyloidosis' is a general term used for a group of diseases where an abnormal protein, called amyloid, accumulates in the tissues. The build-up of amyloid…

Background: Treatment options for patients (pts) with relapsed/refractory (RR) AL amyloidosis are limited. Daratumumab (dara) has been approved as monotherapy (DMT…

Much like multiple myeloma, the treatment landscape of immunoglobulin light chain (AL) amyloidosis is rapidly expanding with novel systemic therapies, says Michael…

Systemic amyloidosis is caused by misfolding and extracellular deposition of circulating proteins as amyloid fibrils, resulting in the dysfunction of vital organs.…