12/23/2025
Some more good news in medicine! Changing lives with new therapies!
The baby saved from a rare disease by a first-ever personalized gene fix has reached a big milestone — taking his first steps ahead of Christmas.
KJ Muldoon was born last year with a genetic disorder called carbamoyl-phosphate synthetase 1 deficiency. Also known as CPS1 deficiency, the metabolic condition affects only 1 in 1.3 million babies and often leads to life-threatening outcomes.
When he was 6 months old, doctors began giving KJ a groundbreaking new treatment — a personalized CRISPR gene editing therapy at Children's Hospital of Philadelphia.
Read more: https://abcnews.visitlink.me/ziYiLb