
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
Let them eat (Taylor Swift) cake: The baker turning A-listers into life-size desserts - 2
Woman leaves bachelorette trip after trusting her gut about sketchy men partying it up with friends - 3
Experience Unrivaled Sound: Top Speakers You Really want to Hear - 4
David Duchovny's new thriller has him stripping down at 65. But its chilling premise hits close to home. - 5
Step into Nature: A Survey of \Handling Trails with Solace\ Climbing Shoes
Iran plans new restrictions in overhaul of Strait of Hormuz rules
Tracking down the Right Equilibrium: Charges versus Personal Costs in Senior Protection.
Top 5 Top of the line Books of the Year
Doctored NXT Summit footage falsely portrays Modi as declaring war on Iran and Pakistan
A definitive Manual for Well known Fragrances
German Court Rejects Bid To Force BMW and Mercedes-Benz To Stop Selling New Combustion-Engine Cars After 2030
Top Pastry: What's Your Sweet Treat of Decision?
Vote in favor of the bloom plan that adds a bit of excellence to your life!
Nexi expands alliance with PayPal













