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Human amyloidosis, still intractable but becoming curable: The essential role of pathological diagnosis in the selection of type‐specific therapeutics

The molecular pathogenesis of human amyloidosis has been elucidated greatly during the last 20 years. Based on the understanding of the molecular mechanisms of amyloid fibril formation and deposition, various kinds of new drugs and therapeutics have been emerging to improve the prognosis of amyloidosis and even cure this disease. In this review article, we first summarize the pathogenesis and state‐of‐the‐art therapeutics of representative types of systemic human amyloidosis, that is, immunoglobulin light chain‐related, transthyretin‐related, amyloid A‐associated and β2‐microglobulin‐related amyloidosis. Next, we describe the essential roles of pathological diagnosis, especially the typing diagnosis of amyloidosis to appropriately guide type‐specific therapies of amyloidosis patients. Finally, we introduce the activities of the government‐funded group for surveys and research of amyloidosis in Japan, especially the nation‐wide pathology consultation system of amyloidosis, which started in April 2018. The nation‐wide improvement of the typing diagnosis of amyloidosis is essential for the appropriate treatment and care of amyloidosis patients in Japan.