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Published on: May 23, 2025
Treatment outcomes and ongoing pediatric trials in steroid-resistant nephrotic syndrome
Chris K Fan1, Heather S Jung1, Yasmine E Pang1
1Division of Pediatric Nephrology, Department of Pediatrics, Emory University School of Medicine, Atlanta, GA, USA.
Insights
New therapies offer hope for children with steroid-resistant nephrotic syndrome (SRNS), a condition with high kidney failure risk. This review covers novel treatments and ongoing clinical trials for pediatric SRNS.
Area of Science:
- Pediatric Nephrology
- Genetics
- Immunology
Background:
- Steroid-resistant nephrotic syndrome (SRNS) in children presents significant challenges, with high rates of treatment resistance and progression to kidney failure.
- Monogenic forms of SRNS and resistance to immunosuppressants pose the greatest risks for pediatric patients.
- Understanding SRNS pathogenesis is crucial for developing targeted therapies.
Purpose of the Study:
- To review contemporary findings from cohort studies on pediatric SRNS.
- To summarize novel therapeutic agents and their specific mechanistic targets.
- To present an overview of recent and ongoing clinical trials for pediatric SRNS.
Main Methods:
- Literature review of contemporary cohort studies.
- Analysis of novel therapeutic agents and their targets in SRNS.
- Compilation of data from recent and ongoing clinical trials.
Main Results:
- Advances in understanding SRNS pathogenesis have led to new targeted therapies.
- Novel agents address genetic, immunologic, and metabolic pathways implicated in SRNS.
- Eight recent and ongoing clinical trials are evaluating these novel approaches.
Conclusions:
- Despite challenges, novel therapies show promise for pediatric SRNS.
- Targeted treatments offer new hope for children at high risk of kidney failure.
- Ongoing clinical trials are essential for advancing pediatric SRNS management.
Abstract:
Despite an increasing number of therapeutic options for pediatric patients with steroid-resistant nephrotic syndrome (SRNS), treatment resistance and risk of progression to kidney failure are still high. Children with monogenic forms of SRNS and resistance to non-steroidal immunosuppressants are at the highest risk. Advances in the understanding of SRNS pathogenesis have enabled the development of novel therapies that target genetic, immunologic, and metabolic mechanisms of disease development and progression. In this review, we summarize select contemporary cohort study findings, novel therapeutic agents and their mechanistic targets, and eight recent and ongoing clinical trials for pediatric patients with SRNS.
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