
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.
NEUESTE BEITRÄGE
- 1
Top 10 Arising Advances That Will Shape What's in store11.08.2023 - 2
Most loved VR Game for Wellness: Which Keeps You Dynamic?01.01.1 - 3
Italy now recognizes the crime of femicide and punishes it with life in prison25.11.2025 - 4
Carnival fever hits Lagos as locals celebrate Afro-Brazilian heritage07.04.2026 - 5
The most effective method to Boost Eco-friendliness in Your Volvo XC4019.10.2023
Ähnliche Artikel
Exploring the Mind boggling Universe of Connections: Individual Bits of knowledge25.09.2023
Guns N' Roses 2026 Tour: How to get tickets, presale times, prices and more01.12.2025
The Reduced Portage Horse: An Inheritance Reconsidered for Present day Experience08.11.2023
5 Instructive Toy Brands for Youngsters05.06.2024
Explora Journeys becomes latest cruise line to be impacted by Middle East war26.03.2026
6 Savvy Locks for Lofts06.06.2024
‘And then we saw the little head.’ Scientists witness rare sperm whale birth30.03.2026
Coffee Prices Finish Higher on Brazil Cop Concerns13.01.2026
What is the 'Survivor 50' Challenge? Hidden immunity idols will be up for grabs in every U.S. state.18.12.2025
Moving Pool Highlights for 202405.06.2024













