Rationale and Design of NEURO-TTRansform, a phase 3 study evaluating the efficacy and safety of AKCEA-TTR-LRx in patients with hereditary transthyretin-mediated Amyloid Polyneuropathy (hATTR-PN)

Tai, L; Obici, L; Giilmore, J; Viney, N; Monteiro, C; Buchele, G; Brambatti, M; Tskimikas, S; Jung, S; O'dea, L; Schneider, E; Geary, R; Monia, B

EUROPEAN JOURNAL OF NEUROLOGY, 2020; 27 (): 514