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Pathogenesis of transthyretin amyloidosis
Amyloid, 2012Current dogma for transthyretin (TTR) pathogenesis is that mutations in TTR alter its structure such that the tetramer becomes unstable and prone to release of monomer which then becomes the putative building block of the fibril. This hypothesis is supported by thermodynamic data showing decreased stability of mutant TTR tetrameric proteins and ...
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Tafamidis for transthyretin amyloidosis
Drugs of Today, 2012Tafamidis meglumine (Vyndaqel®, Pfizer) is a novel, first-in-class drug for the treatment of transthyretin familial amyloid polyneuropathy (TTR-FAP), a rare neurodegenerative disorder characterized by progressive sensory, motor and autonomic impairment that is ultimately fatal.
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La Revue de medecine interne, 2007
Transthyretin amyloidoses are the most common form of amyloidosis. Two different types of transthyretin amyloidoses are described, one is rare, familial, its precursor is the mutated transthyretin, this type is called transthyretin amyloid, the other is more common, its precursor is wild transthyretin, this second type is called senile amyloid.
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Transthyretin amyloidoses are the most common form of amyloidosis. Two different types of transthyretin amyloidoses are described, one is rare, familial, its precursor is the mutated transthyretin, this type is called transthyretin amyloid, the other is more common, its precursor is wild transthyretin, this second type is called senile amyloid.
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RNA-targeting and gene editing therapies for transthyretin amyloidosis
Nature Reviews Cardiology, 2022Alberto Aimo +2 more
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CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis
New England Journal of Medicine, 2021Julian D Gillmore +2 more
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Isolated transthyretin valvular amyloidosis
European Heart Journal, 2023Yi-Tsang Fu, Ling Kuo, Wen-Chung Yu
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Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis
New England Journal of Medicine, 2023Mathew S Maurer +2 more
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Amyloid: the International Journal of Experimental and Clinical Investigation: the Official Journal of the International Society of Amyloidosis, 2023
David Adams +2 more
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David Adams +2 more
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Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
New England Journal of Medicine, 2018Chih-Chao Yang +2 more
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