Patisiran, an RNAi therapeutic for the treatment of hereditary transthyretin-mediated amyloidosis

Kristen, AV; Ajroud-Driss, S; Conceicao, I; Gorevic, P; Kyriakides, T; Obici, L

Kristen, AV (reprint author), Heidelberg Univ, Dept Cardiol, D-69120 Heidelberg, Germany.; Kristen, AV (reprint author), Cardiovasc Ctr Darmstadt, D-64287 Darmstadt, Germany.

NEURODEGENERATIVE DISEASE MANAGEMENT, 2019; 9 (1): 5

Abstract

Hereditary transthyretin-mediated amyloidosis is a rapidly progressive, heterogeneous disease caused by the accumulation of misfolded transthyretin pr......

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