Alpha-1 antitrypsin deficiency • Liver disease

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Investigative possible therapy for first-class RNA interference (RNAi) is promising

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for the treatment of patients with liver disease

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associated with alpha1-antitrypsin deficiency (AAT),

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the results of a phase II study are proposed.

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All 16 patients treated with fazirsiran had a reduction in mutant AAT protein in the liver

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serum, as well as a reduction in globular load, and the patient's liver enzymes

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reached normal levels. university. Rheinisch-Westfälische Technische Hochschule

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Hospital in Aachen, Germany at a meeting of the European Association

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