Cristina Martin-Higueras, Lodovica Borghese, Armando Torres, Fátima Fraga-Bilbao, Raquel Santana-Estupiñán, Constantinos J Stefanidis, Kálmán Tory, Adam Walli, Leire Gondra, Caroline Kempf, Michaela Gessner, Sandra Habbig, Lisa Eifler, Claus P Schmitt, Benjamin Rüdel, Malte P Bartram, Bodo B Beck, Bernd Hoppe
INTRODUCTION: The RNA interference (RNAi) medication lumasiran reduces hepatic oxalate production in primary hyperoxaluria type 1 (PH1). Data outside clinical trials are scarce. METHODS: We report on retrospectively and observationally obtained data in 33 patients with PH1 (20 with preserved kidney function, 13 on dialysis) treated with lumasiran for a median of 18 months. RESULTS: Among those with preserved kidney function, mean urine oxalate (Uox) decreased from 1...
January 2024: KI Reports