
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
The Universe of Impeccable Pearls: A Manual for Valuable Gems - 2
The Conclusive Manual for Spending plan Travel: Opening Undertakings on a Tight budget - 3
5 State of the art Advancements in Computer generated Simulation - 4
Eco-Accommodating Kitchen Machines: 4 Picks for a Manageable Home - 5
Reporter's Notebook: The Post embeds with foreign armies visiting the IDF
Step by step instructions to Pick an Incineration Urn: Variables to Consider
An Extended period of Voyaging Carefully: the World with Reason
The Solution to Individual budget: Dominating Cash The board
Michael Jordan donates $10M to North Carolina medical center in honor of his mother
An Investigate of 6 Creative Specialty Mixed drinks
Really focusing on Succulents: Tips and Procedures
Figure out how to Guarantee Your Dental Embeds Endure forever
From Lounge chair to Money: Online Positions That Will Change Your Profession
2026 Golden Globes: How to watch, start time, TV channel, full nominee list and more













