Primary hyperoxaluria type 1 is a rare genetic disease; the onset of symptoms 
ranges from childhood to the sixth decade of life and the disease may go 
unrecognized for several years. There is an urgent need for drugs able to 
inhibit the liver production of oxalate and to prevent the disease progression; 
lumasiran, an innovative molecule based on RNAi interference, is one of the most 
promising drugs. A group of leading Italian experts on this disease met to 
respond to some unmet medical needs (early diagnosis, availability of genetic 
tests and dosage of plasma oxalate, timing of liver transplantation, need for 
etiologic treatment), based on the analysis of the main scientific evidence and 
their personal experience. Children showing the characteristic symptoms of the 
disease usually undergo a metabolic screening and obtain an early diagnosis, 
while the experience is very limited in adults and the diagnosis difficult. It 
is therefore essential to increase the knowledge around this disease and the 
importance of metabolic and genetic screening to define a checklist of shared 
clinical and laboratory criteria and to establish a multidisciplinary management 
of potential patients. Oxalate is the cause of the disease: it is crucial to 
reduce both oxaluria and oxalemia through appropriate therapeutic strategies, 
able to prevent and/or reduce renal and systemic complications of primary type 1 
hyperoxaluria. Lumasiran allows to significantly reduce the levels of oxalate 
both in blood and in urine, halting the course of the disease and preventing 
serious renal and systemic complications, if the therapy is started at an early 
stage of the disease.