Stunning success in halting Huntington's disease with gene therapy offers new hope to those with the disorder—and potential ...

From cancer biology to proactive health management, customized care is changing patient outcomes, but what are the costs and ...

Life sciences ranked among the top four sectors, with a notable rise of companies integrating AI and biotech in ...

The new therapy is being hailed as a breakthrough. But the small study has some limitations – and hasn’t been peer-reviewed.

A company called uniQure has announced promising results from a trial of a new gene therapy for Huntington's disease. The ...

As well as slowing disease progression by 75 percent at 36 months, the scientists found that neurofilament light protein (NfL)—a biomarker of neuronal damage—levels in spinal fluid were lower in ...

Stanford researchers and their collaborators have revealed a new device that could change the way scientists conduct ...

The first participant in an investigational gene therapy trial of STXBP1 encephalopathy died, Capsida Biotherapeutics said. The death occurred days after dosing, according to information posted on the ...

September is Sickle Cell Awareness Month, and millions of people worldwide are living with the disease. But advances in ...

In a study reported by Uniqure, which developed the experimental gene therapy, scientists found that it slowed progression of ...

A gene therapy injected into patients’ brains slowed progression of the disease by 75 percent, preliminary findings reported.

CDK4/6 inhibitors, combined with hormone therapy, are now the standard treatment for an advanced type of breast cancer; ...