Quality of life outcomes in APOLLO, the phase 3 trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis.

doi:10.1080/13506129.2020.1730790

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Article

Quality of life outcomes in APOLLO, the phase 3 trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis.

Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis - 1 Sept 2020

Obici Laura, Berk John L, González-Duarte Alejandra, Coelho Teresa, Gillmore Julian, Schmidt Hartmut H-J, Schilling Matthias, Yamashita Taro, Labeyrie Céline, Brannagan Thomas H, Ajroud-Driss Senda, Gorevic Peter, Kristen Arnt V, Franklin Jaclyn, Chen Jihong, Sweetser Marianne T, Wang Jing Jing, Adams David

Abstract excerpt

Introduction: Hereditary transthyretin-mediated (hATTR) amyloidosis is a rare, fatal, multisystem disease leading to deteriorating quality of life (QOL). The impact of patisiran on QOL in patients with hATTR amyloidosis with polyneuropathy from the phase 3 APOLLO study (NCT01960348) is evaluated.Methods: Patients received either patisiran 0.3 mg/kg (n = 148) or placebo (n = 77) intravenously once every...

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PMID: 32131641
DOI: 10.1080/13506129.2020.1730790

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