Navigating the Data, Treatments and Breakthroughs of hATTR Amyloidosis

Cancer Cell in human showing abnormal cells.Medical science background concept.

ATTR amyloidosis means A for Amyloid and the TTR is short for the protein “transthyretin.” ATTR is one term that represents different kinds of mutations in a TTR gene that is inherited. That gene mutation makes the transthyretin unstable, so amyloid protein misfolding occurs. The amyloid fibrils then go out into the body and can damage nerves and/or organs, depending on the type of TTR mutation that the patient has inherited. TTR is mainly manufactured in the liver.

The majority of hereditary amyloidosis types are TTR-related, and there are many different variations within ATTR. Most ATTR diseases have a hereditary pattern of organ involvement, approximate age of onset and associated symptoms. It is common that symptoms do not appear until a person is an adult and the degree and severity of illness depends on the individual.

In the past, because ATTR often involves nerve or cardiac involvement, some terms were used when the chemical variations were less defined. Examples of these outdated terms include FAP (Familial Amyloid Polyneuropathy) and FAC (Familial Amyloid Cardiomyopathy).

Today, the different forms of ATTR are termed according to the “chemically based” name of the transthyretin protein variation. An example of this would be ATTRV30M (for ATTR Val30Met), which is the most common ATTR variation.

According to the Amyloidosis Foundation, today’s treatment plans are two-fold:

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