
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
Which Countries Would Suffer Most in a Global Energy Shutdown? This Study Has Answers - 2
Geminid meteor shower 2025 peaks next week. Here's what you need to know about this year's best meteor shower - 3
Minneapolis ICE shooting live updates: Protests continue over agent's killing of Renee Nicole Good; Walz puts National Guard on standby - 4
The Most Encouraging New companies to Look Out For - 5
'Harry Potter' fans rejoice: HBO releases 1st trailer for new TV series, set to premiere this Christmas
Productive CRM Programming for Client Relationship The executives
Catholic influencer shares death of 5-year-old son from 'severe' flu
Addiction-stricken community struggles to keep a syringe program going after Trump's order
Vote In favor of Your Favored Language Interpretation Administration
Eleven Creations And Developments That Steered History
UN chief warns he could refer Israel to ICJ over laws targetting UNRWA
Every year, she thanks the trooper for the arrest that led to her sobriety
15 Outrageous Cosplay Outfits That Will Blow You Away
The Fate of Gaming: 5 Energizing Advancements Not too far off












