
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
German finance minister sees advantages of smartphones in schools - 2
5 Cell phones of the Year - 3
Golden satellite insulation sparkles during test | Space photo of the day for Dec. 30, 2025 - 4
Pick the Ideal Family Feline Variety for Your Home - 5
Venice’s newest marvel is a wild, acrobatic dolphin. His refusal to leave puts him in danger
Untamed life Safe-havens All over the Planet Offering Remarkable Creature Experiences
Understanding the Rudiments of Tree Administrations
Amplifying Cash The executives: The Upsides and downsides of Various Ledgers
Old food pyramid vs. RFK Jr.'s new food pyramid. See what's different.
Vote in favor of your favored spot to peruse
Pleasant Cycle Courses All over the Planet
Tickets for 'Stranger Things' Season 5 finale, to be shown in movie theaters on New Year's Eve, go on sale today. Here's how you can save your seat.
Blood pressure drug recalled for possible cross-contamination
Instructions to Pick the Right Toothbrush for Your Teeth












