#440 - 🔵 [PAS 2026] - Could a Quarterly Injection Replace a Liver Transplant for This Rare Kidney Disease?

Send us Fan MailDr. David Sas, pediatric nephrologist at Mayo Clinic, joins Ben to discuss primary hyperoxaluria type 1 — a rare but devastating genetic disease where the liver overproduces oxalate, flooding the kidneys with crystals and leading to end-stage kidney failure in roughly 60% of patients, historically requiring both a liver and kidney transplant. He presents 60-month long-term extension data on Lumasiran, an siRNA-based therapy that suppresses oxalate production at its source — showing that urinary oxalate drops rapidly within the first three months and stays down with quarterly injections, potentially changing the trajectory of this disease forever. He also issues a direct call to urologists and nephrologists everywhere: if your patient has recurrent calcium oxalate stones, check a 24-hour urine — because primary hyperoxaluria is almost certainly being missed.Support the showAs always, feel free to send us questions, comments, or suggestions to our email: [email protected]. You can also contact the show through Instagram or Twitter, @nicupodcast. Or contact Ben and Daphna directly via their Twitter profiles: @drnicu and @doctordaphnamd. The papers discussed in today's episode are listed and timestamped on the webpage linked below.Enjoy!