PH1 management

Management options for PH1 have traditionally included conservative measures and dual liver and kidney transplantation, but therapeutic approaches are evolving.

Management approaches for PH1 have traditionally included initial measures such as hyperhydration and intensive dialysis to clear oxalate and slow progressive decline in kidney function^1,2. Liver transplantation, which replaces the site of faulty gene expression associated with oxalate overproduction, may provide a potential cure, although there are risks associated with this^1,2. More recent developments include RNA interference (RNAi) therapy, which targets and prevents liver oxalate production, without the need for transplantation³. Current and potential future management approaches for PH1 are discussed below.