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

INTRODUCTION: AKCEA-TTR-L(Rx) is a ligand-conjugated antisense (LICA) drug in development for the treatment of hereditary transthyretin amyloidosis (hATTR), a fatal disease caused by mutations in the transthyretin (TTR) gene. AKCEA-TTR-L(Rx) shares the same nucleotide sequence as inotersen, an antis...

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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Healthcare 2021
Materias:
Study Protocol
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8140170/
https://www.ncbi.nlm.nih.gov/pubmed/33638113
http://dx.doi.org/10.1007/s40120-021-00235-6

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