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Eplontersen, sold under the brand name Wainua, is a medication used for the treatment of transthyretin-mediated amyloidosis. [3] It is a transthyretin-directed antisense oligonucleotide. [3] It was developed to treat hereditary transthyretin amyloidosis by Ionis Pharmaceuticals and AstraZeneca. [4] [5] [6] [7]
In May 2019, the FDA approved two tafamidis preparations for the treatment of transthyretin-mediated cardiomyopathy, but has not approved it for the treatment of transthyretin familial amyloid polyneuropathy. [15] In August 2018, the FDA approved patisiran, an siRNA-based treatment, at an expected cost of up to $450,000 per year. [16]
Treatment depends on the type of amyloidosis that is present. Treatment with high dose melphalan, a chemotherapy agent, followed by stem cell transplantation has shown promise in early studies and is recommended for stage I and II AL amyloidosis. [26] However, only 20–25% of people are eligible for stem cell transplant.
Although not based on a human clinical trial, the only currently accepted disease-modifying therapeutic strategy available for familial amyloid cardiomyopathy is a combined liver and heart transplant. Treatments aimed at symptom relief are available, and include diuretics, pacemakers, and arrhythmia management. Thus, Senile systemic amyloidosis ...
Because transthyretin is made in part by the choroid plexus, it can be used as an immunohistochemical marker for choroid plexus papillomas as well as carcinomas. [citation needed] As of March 2015, there are two ongoing clinical trials undergoing recruitment in the United States and worldwide to evaluate potential treatments for TTR amyloidosis.
Acoramidis is indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. [1] [4] [8] ATTR-CM is a rare and serious disease that affects the heart muscle. [4]
Chemotherapy can treat amyloidosis if it is related to immunoglobulins. [4] Liver transplant can treat amyloidosis if it is related to familial transthyretin. [4]Acoramidis (Attruby) was approved for medical use in the United States in November 2024, to treat adults with cardiomyopathy of wild-type or variant (hereditary) transthyretin-mediated amyloidosis (ATTR-CM) to reduce death and ...
Wild-type transthyretin amyloid (WTTA), also known as senile systemic amyloidosis (SSA), [1] is a disease that typically affects the heart and tendons of elderly people. It is caused by the accumulation of a wild-type (that is to say a normal ) protein called transthyretin .
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