Half of all babies diagnosed with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency die within their first week of life Getty KJ Muldoon was diagnosed with severe carbamoyl phosphate ...

Until recently, even the most advanced gene therapies could only be given after a child was born—often racing against time to prevent irreversible damage. In the first part of this series, we explored ...

Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine Today, the world stands at a crossroads in genetic medicine ...

Editor’s note: On June 3, 2025, KJ Muldoon was discharged from the hospital and is now at home with his family. When a baby born in Philadelphia was announced as the first person to get a gene therapy ...

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of ...

In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...

The US Food and Drug Administration’s (FDA’s) new pathway for gene therapies could be “groundbreaking” if applied broadly, though the extent of its scope remains unclear. In a New England Journal of ...

This is the first time such results have been achieved in both children and adult patients born with a specific type of congenital deafness. When you purchase through links on our site, we may earn an ...

Cell and gene therapies use the body's building blocks - cells and genetic material - to boost the immune system or correct ...

Gene therapy for sickle cell disease may help improve a major contributing factor to stroke risk in patients, reports a new study from St. Jude Children's Research Hospital. Many people with sickle ...

Maoli Duan does not work for, consult, own shares in or receive funding from any company or organization that would benefit from this article, and has disclosed no relevant affiliations beyond their ...

This story is the first in an occasional series following Louisiana’s first patients to receive gene therapy for sickle cell disease. On the third floor of Manning Family Children’s Hospital in New ...