With promise to slow aging and cure some types of cancer, a new gene therapy presents cost and access hurdles for CT patients.

Chantez Sanford Jr., 24, of Southfield, says his life has been transformed after undergoing Lyfgenia gene therapy at Children ...

The movement-related benefits of Itvisma were maintained or continued to deepen over about 15 months after treatment, trial ...

UAB is participating in a Phase I/II trial of AMT 191, a one time gene therapy designed to enable patients with classic Fabry disease to produce their own missing enzyme, potentially reducing the need ...

A single dose of experimental gene therapy GS001 reduced bleeding episodes in men with severe hemophilia A, a study showed.

Care hurdles now add to the disease burden faced by U.S. sickle cell patients, despite the promise of emerging options like gene therapy.

Ultragenyx Pharmaceutical said on Thursday its experimental gene therapy helped patients in a ​late-stage trial to better control ammonia levels linked to ‌a rare inherited disorder.

A team of University of Kentucky researchers has uncovered a surprising clue in the battle against Alzheimer's disease that could help doctors predict, and ultimately prevent, a common side effect of ...

Radiotherapy is a key foundation of oncologic treatment that is used across the spectrum of cancer indications. Advances in imaging, treatment planning, and dose delivery have led to increasingly ...

Gene editing technologies have rapidly evolved from experimental tools into promising therapeutic strategies in oncology. Advances in programmable nucleases, epigenome-targeting approaches, and ...

ATLANTA — Researchers at Georgia State University say they may have found a way to prevent two main side effects associated with popular GLP-1 weight loss drugs. Those side effects include muscle loss ...

The Food and Drug Administration Monday unveiled the details of a new policy designed to make it easier and quicker for patients with very rare diseases to get cutting-edge treatments. The new ...