[Editorial] Learning from a decade of progress in amyloidosis

Improving outcomes for people with rare diseases is a major challenge, but it is possible.

Amyloidosis is a heterogeneous group of rare disorders in which amyloid (misfolded protein deposits) accumulates in the tissues, causing progressive organ failure.

Until recently, it was a rapidly fatal disease with few therapeutic options.

However, as detailed in a Review in this issue, several new treatments have been approved since 2018, and for people with some subtypes estimated 10-year survival has increased from about 5% to 20%. ...