
Squawk Pod BONUS The Path with Becky Quick: Head of the Class 3/20/26
Mar 20, 2026
Kyle Muldoon, father who lived his son’s diagnosis and hospital journey, and Nicole Muldoon, mother who navigated care and the choice for personalized therapy. They recount life-threatening neonatal complications, a transfer to CHOP, the family’s decision to pursue experimental personalized gene editing, infusion day, early signs of improvement, leaving the hospital after 307 days, and hopes for broader rare-disease research.
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Birth Crisis And Immediate ICU Transfer
- Nicole and Kyle Muldoon described becoming parents to KJ, born with severe CPS1 deficiency that left him hours-old with a nonworking urea cycle and ammonia in the 1600s.
- They recounted emergency C-section, eight-hour maternal surgery, and KJ rushed to CHOP for dialysis and intensive care as the family scrambled to keep hope alive.
Refusing Immediate Comfort Care To Give KJ A Chance
- Doctors presented options including comfort care and transplant, but Nicole and Kyle refused immediate hospice and chose to give KJ a chance to fight while remaining open to readdressing later.
- Nicole emphasized writing contingency letters pre-surgery and deciding not to make a hasty end-of-life choice while still learning prognosis details.
Personalized Base Editing Built On Extensive Dress Rehearsals
- CHOP clinicians developed a personalized base editing approach they had practiced for over a year before treating KJ, enabling the first-in-human personalized CRISPR-based therapy.
- Dr. Rebecca Ahrens‑Nicholas and team rehearsed protocols on urea cycle disorder models, then applied tailored lipid‑packaged IV doses targeting KJ's specific DNA misspelling.
