Lumasiran is a small interfering RNA used in the treatment of primary hyperoxaluria type 1 (PH1). This condition, caused by a deficiency in the enzyme alanine-glyoxylate aminotransferase, leads to an accumulation of oxalate, causing calcium crystal formation. These patients experience frequent kidney stones, nephrocalcinosis, and renal failure.
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Lumasiran is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary and plasma oxalate levels in pediatric and adult patients.
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