Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data
Abstract Aims Amyloidogenic transthyretin (ATTR) amyloidosis is a fatal disease characterized by progressive cardiomyopathy and/or polyneuropathy. AKCEA‐TTR‐LRx (ION‐682884) is a ligand‐conjugated antisense drug designed for receptor‐mediated uptake by hepatocytes, the primary source of circulating...
Main Authors: | Nicholas J. Viney, Shuling Guo, Li‐Jung Tai, Brenda F. Baker, Mariam Aghajan, Shiangtung W. Jung, Rosie Z. Yu, Sheri Booten, Heather Murray, Todd Machemer, Sebastien Burel, Sue Murray, Gustavo Buchele, Sotirios Tsimikas, Eugene Schneider, Richard S. Geary, Merrill D. Benson, Brett P. Monia |
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Format: | Article |
Language: | English |
Published: |
Wiley
2021-02-01
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Series: | ESC Heart Failure |
Subjects: | |
Online Access: | https://doi.org/10.1002/ehf2.13154 |
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