
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
Fuel Price Spike Drives Surge in Used EV Sales in Europe - 2
Mount Everest Climbers 'Poisoned' by Guides Prompting Mass Helicopter Rescues in $20 Million Insurance Fraud Scheme, Police Say - 3
Defense Minister Katz moves to extend IDF service to 36 months - 4
Couch Styles of 2024: What's Moving - 5
Banks for High Fixed Store Rates: Amplify Your Reserve funds
My Excursion to Monetary Autonomy: Awesome ways to save cash
Grass Care Administrations for a Wonderful, Sound Yard
Purchases of iPhone 17 Pro soar across Gaza amid 'limited' humanitarian aid
What do scientists hope to learn from NASA's historic Artemis 2 moon flyby?
Wolf bites woman in a shopping area in Germany's 2nd-biggest city
Top 15 Style Creators Changing the Business
Rocket shines under the northern lights | Space photo of the day for March 25, 2026
Travel Through France's Most Iconic Wine Regions By Train On An Immersive Seven-Day Journey
How to watch the 2025 Macy's Thanksgiving Day Parade for free













