New therapeutic hope for rare podocytopathies

Podocytopathies are kidney diseases caused by podocyte injury or dysfunction that drives proteinuria or nephrotic syndrome. A significant expansion in understanding of the complex causes and mechanisms of podocyte injury, as well as potential therapeutic approaches, has been achieved over the past two decades with rapid advances in genomics in both research and clinical practice. Podocytopathies associated with monogenic mechanisms account for approximately 30% of cases with steroid-resistant nephrotic syndrome (SRNS) or focal segmental glomerulosclerosis lesions (FSGS), with even higher proportions in pediatric patients. Podocytopathies related to each gene mutation are rare. However, recent developments in these rare podocytopathies contribute to an optimistic outcome for the future to eventually avoid kidney failure. This review therefore focuses on aspects of new advances of therapies in rare podocytopathies with monogenic causes.