Lumasiran, Isolated Kidney Transplantation, and Continued Vigilance
Patient 1, a girl with infantile oxalosis, was homozygous for c.731T→C (of unclear predicted pyridoxine sensitivity). She underwent intensive dialysis for 10 months before the initiation of lumasiran treatment, followed by 13 months of intensive dialysis plus lumasiran. At 3 years of age, she underwent isolated kidney transplantation from a deceased donor; the plasma oxalate level at the … Leggi tutto