
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
'Supergirl' drops 1st teaser trailer: Watch Milly Alcock as Kara Zor-El and the return of Krypto the Superdog - 2
Figure out How to Plan for Your Web-based Degree monetarily - 3
The 15 Most Powerful Forerunners in Business - 4
6 Natural products High In Vitamins,Which One Do You Like to Eat - 5
Most loved Well known Accessory Styles For 2024
Help Your Business with Master Web based Promoting Arrangements
Don’t let food poisoning crash your Thanksgiving dinner
Over 60 local leaders push Netanyahu to halt haredi draft bill, warn of social rift
Winter storm headed for Midwest to Northeast. Here's how much snow to expect.
One spent $20 on candy. Another paid $700 for a custom costume. Here's how Halloween costs stacked up this year.
Getting Your Youngsters' Future: Grasping Legacy Regulations
Sean 'Diddy' Combs faces new sexual assault allegations, currently under investigation by Los Angeles Sheriff's Department
41 Young Men Die in South Africa After Circumcision Initiation
Step by step instructions to Boost the Eco-friendliness of Your Smash 1500.











