A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial ...

Groundbreaking Phase 1/2a clinical trials co-led by Linda Laux, MD, from Ann & Robert H. Lurie Children's Hospital of Chicago ...

Some genetic mutations that are expected to completely stop a gene from working surprisingly cause only mild or even no symptoms. Researchers in ...

The first gene-regulating treatment for epilepsy achieves promising results in an initial trial with 81 patients ...

A team of researchers at Texas Children's Duncan Neurological Research Institute (NRI) and Baylor College of Medicine report ...

Atavistik Bio is using the cash to develop “allosteric” medicines it believes can offer precise impact on diseases like ...

Casey Means says Americans share a single underlying condition.

All complex biological systems—like the DNA, RNA and proteins constantly being copied and built within our cells—are prone to errors. That means as life evolved to be more elaborate, it also had to ...

Affecting roughly half a million Americans each year, bacterial infections caused by Clostridioides difficile—commonly known ...

The FDA approved a vaccine for mumps nearly 60 years ago, so why are some areas of the United States experiencing outbreaks now, and who should be concerned?