Introduction to FSGS

In this episode of the Rare Kidney Disease Show, Dr. Howard Trachtman, Adjunct Professor of Pediatrics at the University of Michigan, and Dr. Chris Gisler, Medical Director at Travere Therapeutics, explore the complexities of FSGS, covering its pathophysiology, classifications, and clinical presentation.

They discuss key drivers of kidney failure, challenges in diagnosis and management, and the unmet need for safe and effective treatments.

Listeners will gain insights into the patient journey, role of precision medicine, disease heterogeneity, and the future of FSGS clinical trial design. The episode concludes with how PARASOL is shaping advancements in FSGS research and clinical trials.

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Episode Summary

“A lot of our community has really highlighted the pivotal role of the podocyte in the maintenance of glomerular barrier function.” (7:52)

“I would dread the conversation when I had to tell them their child had a diagnosis of FSGS because one has to relay […] the likelihood, the significant likelihood of progression to kidney failure.” (14:16)

“The heterogeneity is what’s made it very difficult for clinicians to really be able to define a rational approach to this disease.” (24:44)

Evolving Classification of FSGS – Once defined primarily by histopathology, FSGS is now recognized as a heterogeneous disorder with distinct genetic, immune, and metabolic etiologies. This shift has critical implications for diagnosis and treatment.
FSGS and the Role of Podocyte Injury – Podocyte loss is a key driver of FSGS progression, with genetic, immune, and metabolic insults leading to irreversible damage. Understanding this mechanism is crucial for developing targeted therapies.
Challenges in Treatment and Therapeutic Gaps – Current treatment strategies rely on empiric use of steroids and calcineurin inhibitors, with variable efficacy and significant side effects. A more targeted approach is needed to improve patient outcomes.

Disclaimer: Guest speakers of the Rare Kidney Disease Show may be paid consultants of Travere Therapeutics.

MA-SP-25-0033

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