Inotersen to Treat Polyneuropathy Associated with Hereditary Transthyretin (hATTR) Amyloidosis by Christopher Robinson, Cynthia Pham, Alec M. Zamarripa, Chase S. Dugay, Christopher A. Lee, Amnon A. Berger, Avi Landman, Elyse M. Cornett, Hisham Kassem, Alan D. Kaye, Ivan Urits, Omar Viswanath, Latha Ganti

“…# Background Amyloidosis is a group of diseases with the common pathophysiology of protein misfolding and aberrant deposition in tissue. There are both acquired and hereditary forms of this disease, and this review focuses on the latter hereditary transthyretin-mediated (hATTR). hATTR affects about 50,000 individuals globally and mostly appears as one of three syndromes - cardiac, polyneuropathy, and oculoleptomeningeal. …”
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