Hereditary (familial) amyloid transthyretin-related (hATTR) amyloidosis is a rare, severely debilitating, and often fatal disorder caused by a mutation in the gene encoding transthyretin (TTR). It results in abnormal amyloid protein buildup in multiple organ systems, leading to progressive peripheral neuropathy, cardiomyopathy, nephropathy, and gastrointestinal dysfunction. Due to the multiorgan system involvement of the disease, an interdisciplinary approach to treatment is crucial to manage all comorbidities. Available treatment modalities include surgical and pharmacotherapeutic options.
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Cite this: Jefferson R. Roberts, Victoria M.F. Mank. Fast Five Quiz: Treatment of Hereditary (Familial) Amyloid Transthyretin-Related (hATTR) Amyloidosis - Medscape - May 24, 2021.