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2018•Hospital PharmacyOpen access

New Medications in the Treatment of Hereditary Transthyretin Amyloidosis

Scot Walker

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Abstract

Hereditary transthyretin amyloidosis is an inherited disorder that results in the gradual progressive deposit of abnormal protein called amyloid in the body's organs and tissues. There are currently no approved drugs to treat transthyretin amyloidosis, and patients may require liver transplantation for survival. There are a few drugs in development to treat hereditary transthyretin amyloidosis either by stabilizing the abnormal protein or by decreasing production of transthyretin. Both methods are being developed to slow progression of the disease.

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What this paper is about

Hereditary transthyretin amyloidosis is an inherited disorder that results in the gradual progressive deposit of abnormal protein called amyloid in the body's organs and tissues. There are currently no approved drugs to treat transthyretin amyloidosis, and patients may require liver transplantation for survival. There are a few drugs in development to treat hereditary transthyretin amyloidosis either by stabilizing the abnormal protein or by decreasing production of transthyretin. Both methods are being developed to slow progression of the disease.

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Available abstract

Hereditary transthyretin amyloidosis is an inherited disorder that results in the gradual progressive deposit of abnormal protein called amyloid in the body's organs and tissues. There are currently no approved drugs to treat transthyretin amyloidosis, and patients may require liver transplantation for survival. There are a few drugs in development to treat hereditary transthyretin amyloidosis either by stabilizing the abnormal protein or by decreasing production of transthyretin. Both methods are being developed to slow progression of the disease.

Key concepts: Transthyretin, Medicine, Amyloidosis, Amyloid (mycology), Amyloid fibril, Liver transplantation, Disease, Amyloid disease

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