Article
Tafamidis for transthyretin amyloidosis.
Drugs of today (Barcelona, Spain : 1998) - 1 May 2012
de Lartigue J
Abstract excerpt
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. Pathogenic mutations in the transthyretin (TTR) protein lead to destabilization of its tetrameric structure and subsequent formation of...
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