
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
6 Travel Services for Colorful Get-aways: Pick Your Fantasy Escape - 2
Roche breast cancer pill cuts risk of disease recurrence by 30% in trial - 3
Rocket shines under the northern lights | Space photo of the day for March 25, 2026 - 4
Don't plan to cook on Thanksgiving? Here are the restaurants and fast food places that are scheduled to be open - 5
The Magnificence of Do-It-Yourself Skincare: Regular Recipes and Tips
Director of Swiss hospital describes the rush to treat the injured from Alpine resort bar fire
Instructions to Really Oversee Unsold SUVs in the Auto Business
At least 36 dead in major fire in Hong Kong residential blocks
Step by step instructions to Prepare with Senior Protection for Inward feeling of harmony.
Why some African countries are prone to military takeovers
I'm a woman who's into weightlifting. Was I man enough for the creatine-packed 'Man Cereal'?
The most effective method to Boost Eco-friendliness in Your Volvo XC40
Solar storms have influenced our history – an environmental historian explains how they could also threaten our future
A definitive Manual for Choosing Indoor Plants Ideal for Your Space













