Patisiran sodium

• CAT Number: I045362
• CAS Number: 1386913-72-9
• Molecular Weight: 13427.10 (AS: 6661.3; SS: 6765.8)
• Purity: 95%
• Target: TTR
• Target Protein: P02766,,,
• Appearance: Solid
• Storage: Dry, dark and at 2 - 8 °C for six months or -20°C for two years.

Patisiran sodium（Cat No.:I045362）is an RNA interference (RNAi) therapeutic used for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis. It targets and silences the transthyretin (TTR) gene, reducing the production of misfolded TTR protein responsible for amyloid deposits in tissues. By lowering TTR protein levels, Patisiran helps alleviate the debilitating symptoms of hATTR, including neuropathy and cardiomyopathy. Administered via intravenous infusion, it represents a groundbreaking treatment for a previously untreatable genetic disorder. Its targeted mechanism provides significant improvements in quality of life for patients with this progressive disease.

Reference

[1]. Adams D, et, al. Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis. N Engl J Med. 2018 Jul 5;379(1):11-21.
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[2]. Hoy SM. Patisiran: First Global Approval. Drugs. 2018 Oct;78(15):1625-1631.
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[3]. Kristen AV, et, al. Patisiran, an RNAi therapeutic for the treatment of hereditary transthyretin-mediated amyloidosis. Neurodegener Dis Manag. 2019 Feb;9(1):5-23.
[Content Brief]