Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-L(Rx) (ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
针对遗传性转甲状腺素蛋白介导的淀粉样多发性神经病,开展反义寡核苷酸AKCEA-TTR-L(Rx) (ION-682884-CS3)的全球3期NEURO-TTRansform研究的设计和原理
期刊:Neurology and Therapy
影响因子:4.8
doi:10.1007/s40120-021-00235-6
Coelho, Teresa; Ando, Yukio; Benson, Merrill D; Berk, John L; Waddington-Cruz, Márcia; Dyck, Peter J; Gillmore, Julian D; Khella, Sami L; Litchy, William J; Obici, Laura; Monteiro, Cecilia; Tai, Li-Jung; Viney, Nicholas J; Buchele, Gustavo; Brambatti, Michela; Jung, Shiangtung W; St L O'Dea, Louis; Tsimikas, Sotirios; Schneider, Eugene; Geary, Richard S; Monia, Brett P; Gertz, Morie