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Synonyms: AD-65585 | ALN-65585 | ALN-G01 | Oxlumo®
lumasiran is an approved drug (EMA, FDA & UK MHRA (2020))
Compound class:
Nucleic acid
Comment: Lumasiran is a double-stranded small interfering ribonucleic acid (siRNA) drug. It targets HAO1 mRNA as a mechanism to reduce the hepatic oxalate overproduction that is responsible for disease pathology in patients with primary hyperoxaluria type 1 (PH1) [1]. We have been unable to locate a full chemical SMILES for the drug, or its HELM notation.
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| No information available. |
Summary of Clinical Use  |
| Lumasiran is an approved treament for PH1. |
| Clinical Trials | |||||
| Clinical Trial ID | Title | Type | Source | Comment | References |
| NCT03681184 | A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1 | Phase 3 Interventional | Alnylam Pharmaceuticals | ||
| NCT03905694 | A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1 | Phase 3 Interventional | Alnylam Pharmaceuticals | ||
| NCT04152200 | A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 | Phase 3 Interventional | Alnylam Pharmaceuticals | ||
