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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 ...
Cardiac amyloidosis is a subcategory of amyloidosis where there is depositing of the protein amyloid in the cardiac muscle and surrounding tissues. Amyloid, a misfolded and insoluble protein, can become a deposit in the heart's atria, valves, or ventricles .
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 .
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.
Tafamidis was approved in the European Union in 2011 for the treatment of transthyretin amyloidosis with polyneuropathy, and in Japan in 2013. [6] [17] In the United States, it was rejected for the treatment of transthyretin amyloidosis with polyneuropathy because the Food and Drug Administration saw insufficient evidence for its efficacy. [20] [7]
Acoramidis, sold under the brand name Attruby, is a medication used for the treatment of cardiomyopathy. [1] It is a near-complete (>90%) transthyretin stabilizer, developed to mimic the protective properties of the naturally-occurring T119M mutation, [4] [5] to treat transthyretin amyloid cardiomyopathy. It is taken by mouth. [1]
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