
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
Many European nations want Israel to cancel 19 new settlement plans - 2
8 Fundamental Stages: Novice's Manual for Secure Your Android with a VPN - 3
Famous SUVs With Low Energy Utilization In 2024 - 4
The Following Huge Thing: 5 Progressive Tech New businesses - 5
Cuba fights to contain spread of mosquito-borne chikungunya virus
Cyclone causes blackout, flight chaos in Brazil's Sao Paulo
Picking the Right Air Purifier for Your Home
Pick Your Favored method of transportation
6 Popular Ladies' Aromas On the planet
Mont Blanc road tunnel reopens to traffic after 15 weeks of repairs
Day to day Temporary Positions That Compensate Fairly in the US
Amid growing bipartisan scrutiny of Pete Hegseth, Trump says he 'wouldn't have wanted … a second strike' on alleged Venezuelan drug boat survivors
Witness the elegance of the cosmic butterfly in a remarkable telescope photo
The most effective method to Guarantee Thorough Inclusion in Senior Protection.













