
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
Invigorating Spots To Go Kayaking All over The Planet - 2
Iran begins cloud seeding to induce rain amid historic drought - 3
Bennu asteroid samples provide clues about solar system origins and 'space gum' - 4
Best Wellness Tracker Keep You On target - 5
Reconnecting with an old friend is a story of distance, loss and rediscovery
Picking the Right Home Machines: A Commonsense Aide
'Euphoria' releases Season 3 trailer, premiere date: Watch Rue and Laurie finally face off
Real time features for Films and Programs
Vote In favor of Your #1 Compelling Female Producer
Winter storm headed for Midwest to Northeast. Here's how much snow to expect.
Lawsuit claims ChatGPT exacerbated man's delusions leading to murder-suicide
Anthony Joshua's driver charged over Nigeria crash that killed two
Novo Nordisk cuts Wegovy price as CEO pledges to go 'all in' on weight loss pill
Which Switch Game Do You Suggest? Share Your Decision












