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New method reveals how mutations drive transthyretin amyloidosis and guides precision drug design

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An international research team reveals new molecular mechanisms associated with pathogenic mutations in the protein transthyretin that cause transthyretin amyloidosis (ATTR), a group of fatal progressive diseases. The results, obtained thanks to a new methodological approach, open the door to the development of drugs with higher therapeutic potential, designed specifically for the variants of the protein associated with the disease.














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