Pancreatitis is a complex inflammatory disease driven by both genetic and environmental factors. It poses substantial ...

Elin Lewis, a 12-year-old girl from Greenwood, Indiana, has become the first patient in the state to receive an FDA-approved ...

In a worldwide first, a one-and-done gene therapy has been approved to treat a form of hereditary deafness. Many of the ...

A breakthrough FDA-approved treatment is changing how genetic deafness is treated.

Heart disease remains the leading cause of death in the United States, responsible for about one in every five deaths each ...

Novel CRISPR-Cas9 treatments are proving effective in reversing disease for life. But ensuring their long-term safety will ...

Encoded Therapeutics gives a heads-up to other gene therapy company about the trade-offs with a drug meant to improve safety.

Dr. Marty Makary noted it had "record speed approval," the fastest approval of any gene therapy device combination in U.S. history.

Genetic sleuthing uncovers a rare case of cancer caused by a virus administered as part of a child’s treatment for a genetic ...

Doctors have linked a boy's tumor to a gene therapy, but say the rare risks need to be weighed against the profound benefits ...

A planned first-in-human trial aims to treat GM1 gangliosidosis in utero, possibly laying the foundation for prenatal gene ...

A group of Queen’s researchers is hoping to use gene therapy and new grant money to cure a handful of rare, usually fatal ...