ABOVE: A newly approved RNAi-based therapy reduces the overproduction of oxalate among people who have a rare genetic disorder. The condition leads to kidney stones, which are formed by calcium oxalate crystals such as these, and tissue damage.
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Last month, the US Food and Drug Administration approved lumasiran (Oxlumo), a drug to treat a rare genetic disorder known as primary hyperoxaluria type 1. PH1 results in an overproduction of a substance known as oxalate by the liver, which can accumulate in the kidney and urinary tract and cause a wide range of effects, including kidney stones, widespread organ damage, and end-stage renal disease.
The only cure for PH1 is a liver transplant. Lumasiran, which was developed by Cambridge, Massachusetts-based Alnylam Pharmaceuticals, is the first drug that reduces oxalate levels in patients, thus reducing the risk of complications later in life. “This is really the first treatment that medically ...
