
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
AfD faction in western Germany ousts councilman for firebrand speech - 2
Christmas 2025 skywatching guide: What you can see in the night sky on Dec. 25 - 3
Explainer-What will change with the US reclassification of marijuana? - 4
Shadow Cats: The Elusive Leopards Surviving Against Impossible Odds - 5
Meet the Stars of the Feline World: Well known Pet Feline Varieties
Vote In favor of Your Favored Kind Of Attire
Ministry: New German petrol price regulation takes effect on April 1
What to know about King Charles III's cancer treatment and his message to the public
Reality TV star Spencer Pratt, who lost his home in Palisades Fire, is running for mayor of Los Angeles
Step by step instructions to Prepare with Senior Protection for Inward feeling of harmony.
Abbott issues US device correction for some glucose monitors over faulty readings risk
Kissing is an ‘evolutionary conundrum.’ Scientists just mapped its unexpected origins
Flu season is underway. What are common symptoms to watch for?
Posts falsely claim Netanyahu video fabricated to cover up his death













