BridgeBio

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.…

The disease causing agent in systemic AL amyloidosis is a monoclonal immunoglobulin free light chain, or fragments thereof, circulating in the blood. It is not…

The amyloidoses are a group of protein-folding disorders in which >=1 organ is infiltrated by proteinaceous deposits known as amyloid. The deposits are derived…

AL amyloidosis is treated with the same set of chemotherapy drugs that are used to treat multiple myeloma. However, it is important for patients with AL amyloidosis…