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Synonyms: ALN-65492 | ALN-TTRSC02 | Amvuttra® | votrisiran
vutrisiran is an approved drug (EMA, FDA & UK MHRA (2022))
Compound class:
Nucleic acid
Comment: Vutrisiran (ALN-65492) is a GalNAc conjugated double-stranded small interfering ribonucleic acid (siRNA) drug. It acts as a transthyretin (TTR) synthesis inhibitor.
We have been unable to locate a full chemical SMILES for the drug, or its HELM notation. |
| References |
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1. Adams D, Tournev IL, Taylor MS, Coelho T, Planté-Bordeneuve V, Berk JL, González-Duarte A, Gillmore JD, Low SC, Sekijima Y et al.. (2023)
Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial. Amyloid, 30 (1): 1-9. [PMID:35875890] |
