Federal Grant Award Summary

The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) awarded Boston Children's Hospital a Project Grant totaling $893,823 under the Diabetes, Digestive, and Kidney Diseases Extramural Research program (CFDA 93.847) to conduct genomically-driven discovery research on pediatric steroid-sensitive nephrotic syndrome (SSNS). The research initiative, which commenced February 1, 2026, and concludes December 31, 2030, aims to advance the molecular understanding of SSNS—the most common pediatric nephrotic syndrome subtype—through genetic studies and genome-wide association studies (GWAS). Building on a recent global GWAS identifying 11 risk loci in a cohort of 2,440 cases, the research will characterize the polygenic architecture of SSNS, identify genetic subtypes for improved patient classification, and support the development of biomarkers and targeted therapeutics to enable more precise treatment approaches and reduce the chronic immunosuppressive burden currently imposed on affected children.

The project addresses a critical clinical need to move beyond nonspecific histologic and treatment response classifications of nephrotic syndrome toward genetically-informed precision medicine. By elucidating immune-related and other pathobiological mechanisms underlying SSNS, the research will provide a foundation for developing interventions that minimize the significant morbidity associated with long-term corticosteroid and immunomodulating therapy, including infectious risks, hypertension, osteoporosis, infertility, and cataracts, while potentially reducing recurrence rates in transplanted kidneys among children who progress to end-stage kidney disease.