A new class of life-saving drugs is helping children who once had no hope. But some carry a price tag of millions for a ...

Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike to ...

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 ...

Advancements in genetics have already begun changing the way scientists study, predict, diagnosis and treat diseases. But how does the public feel about gene therapy and gene editing? In general, the ...

Researchers in the lab of Hans-Peter Kiem, MD, PhD, at Fred Hutch Cancer Center have devised a method that could one day treat genetic hematologic disorders by correcting how the body makes blood ...

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 ...

Lowering cholesterol is one of the most effective ways to reduce your risk of heart disease, and it may soon be possible to get a one-and-done gene therapy to keep cholesterol and triglyceride levels ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.

After Pfizer let limited patient interest get the best of its hemophilia B gene therapy ambitions and discontinued Beqvez, CSL Behring naturally faced the question of whether it can hold its ground ...

Hemophilia gene therapies arrived a few years ago with blockbuster expectations. But now, one therapy has been pulled from ...

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 ...