
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 is walking and getting ready to celebrate the holiday season at home with his parents and three siblings.
KJ 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.
Doctors save baby's life with first-ever gene fix for deadly rare disease
The gene-editing therapy works like a "molecular spell-checker,” finding and fixing the specific genetic error causing his condition.
After three infusions and after spending the first 10 months of his life in a hospital, KJ was discharged and sent home in June.
Baby saved by gene-editing therapy 'graduates' from hospital, goes home
Since then, KJ has continued to grow and thrive with his family, celebrating his first birthday at home and taking part in one of the family’s favorite rituals -- cheering for their beloved Philadelphia Eagles.
Doctors hope new technology like CRISPR gene-editing therapies can be the key to treating more than 7,000 rare diseases, such as sickle cell disease, cystic fibrosis, Huntington’s disease and muscular dystrophy, that affect approximately 30 million people across the country.
LATEST POSTS
- 1
Human evolution’s biggest mystery has started to unravel. How 2025 tipped the scales - 2
Cannabis reclassification could 'open the floodgates' for research, scientists say - 3
Figure out How to Augment the Advantages of a Web-based Degree - 4
Signature Scents: A Manual for Outstanding Fragrances - 5
Vaccine exemptions for religious or personal beliefs are rising across the U.S.
Uncover the Manageable Fish Practices: Sea agreeable Feasting
Which Film Has the Incomparable Melodic Score?
Pick Your Favored method of transportation
Chemical leak in Oklahoma forces evacuations and leaves many ill
Venice’s newest marvel is a wild, acrobatic dolphin. His refusal to leave puts him in danger
Sydney Sweeney's American Eagle campaign and Kendrick Lamar's Super Bowl performance were among the 10 biggest pop-culture moments of 2025
NASA astronauts take new moonsuit for a swim | Space photo of the day for Nov. 28, 2025
Hypothermia claims newborn in Gaza and more babies are at risk, doctor says
Father and son spending Christmas together after health scares












