Results 181 to 190 of about 16,120 (219)
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Tafamidis for transthyretin amyloidosis

Drugs of Today, 2012
Tafamidis 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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An aggressive form of transthyretin amyloidosis

Amyloid, 2017
In 1990, Ueno et al. identified a new transthyretin (TTR) mutation in a 45-year-old Japanese man with peripheral neuropathy of six-years duration [1].
Noel R, Dasgupta   +3 more
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Pathogenesis of transthyretin amyloidosis

Amyloid, 2012
Current 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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Renal Involvement in Transthyretin Amyloidosis: The Double Presentation of Transthyretin Amyloidosis Deposition Disease

Nephron, 2022
Transthyretin (TTR) amyloidosis (ATTR) is either an inherited condition or a non hereditary disease due to misfolding of wild-type (WT) TTR. Amyloid deposits can be mainly detected in nerves in the inherited form and in myocardium in the acquired variant.
Roberta Fenoglio   +7 more
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Hereditary transthyretin-related amyloidosis

Acta Neurologica Scandinavica, 2018
Hereditary transthyretin(TTR)-related amyloidosis (ATTRm amyloidosis) is an endemic/non-endemic, autosomal-dominant, early- and late-onset, rare, progressive disorder, predominantly manifesting as length-dependent, small fiber dominant, axonal polyneuropathy and frequently associated with cardiac disorders and other multisystem diseases.
Josef Finsterer   +5 more
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Expanding the spectrum of transthyretin amyloidosis

Muscle & Nerve, 2019
See article on pages 95–100 in this issue.
Haruki Koike, Masahisa Katsuno
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Laboratory Assessment of Transthyretin Amyloidosis

Clinical Chemistry and Laboratory Medicine, 2002
Mutations in transthyretin (TTR) are the most common cause of autosomal dominant systemic amyloidosis. To date, more than 80 TTR mutations have been associated with amyloidosis in humans. A high prevalence of some mutations like Val122Ile which is identified in 3% of African Americans indicates the necessity of thorough investigation of patients ...
Merrill D, Benson   +2 more
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Clusterin regulates transthyretin amyloidosis

Biochemical and Biophysical Research Communications, 2009
Transthyretin (TTR) is a human disease-associated amyloidogenic protein that has been implicated in senile systemic amyloidosis (SSA) and familial amyloidotic polyneuropathy (FAP). FAP typically results in severe and early-onset disease, and the only therapy established so far is liver transplantation; thus, developing new strategies for treating FAP ...
Ko-Woon, Lee   +9 more
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Rate of Progression of Transthyretin Amyloidosis

The American Journal of Cardiology, 2011
Hereditary transthyretin (TTR) amyloidosis is an adult-onset disease characterized mainly by peripheral neuropathy and cardiomyopathy. Although disease progression is usually 5 to 15 years from time of diagnosis to death, no specific measurements of disease progression have been identified.
Merrill D, Benson   +5 more
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Vutrisiran for Transthyretin Amyloidosis Cardiomyopathy

Current Vascular Pharmacology

Angelica Lehker, Debabrata Mukherjee
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