
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
Want to be better about saving money in 2026? Try these money-saving tips for having a ‘low-buy’ January and beyond - 2
Manual for 6 Hot Brilliant Beds - 3
She was the supermodel dubbed 'The Face' in the '80s. Joining OnlyFans in her 60s taught her a lot. - 4
The Electric Bicycle Americans Can Confide in 2024 - 5
Getting Your Youngsters' Future: Grasping Legacy Regulations
OpenAI launches ChatGPT Health to connect medical records, wellness apps
Exposure to neighborhood violence leads some Denver teens to use tobacco and alcohol earlier, new study shows
Figure out how to Team up with Your Auto Crash Legal advisor for Best Outcomes
An Extended period of Voyaging Carefully: the World with Reason
10 Demonstrated Systems to Develop Your Internet based Business
No red, no long shorts: The fashion rules Joe Burrows lives by
See a half-lit moon shine among the stars of Aquarius on Nov. 27
The most effective method to Pick the Ideal Shrewd Bed for Your Special Rest Needs
Cygnus XL brings cargo to the ISS for 1st time | Space photo of the day for Dec. 1, 2025













