A heart attack survivor is the first person to receive groundbreaking gene therapy which aims to prevent a major heart procedure from failing ...

The U.S. Food and Drug Administration (FDA) has chosen Dr. Vinay Prasad, a professor at the University of California-San Francisco, to lead its Center for Biologics Evaluation and Research. An ...

AVALA Hospital, in partnership with Retina Associates New Orleans, has successfully performed Louisiana’s first ENCELTO procedure, a cell-based gene therapy for adults with macular telangiectasia type ...

For those born with certain types of congenital deafness, the cochlear implant has been a positive and enabling technology.

Scientists from Johns Hopkins Medicine and eight other institutions in the United States, Africa and Europe say they have identified a potential new gene target that could be edited to treat sickle ...

Onasemnogene abeparvovec-brve is the first gene replacement therapy approved for SMA patients aged 2 years and older, expanding access beyond infants. Phase 3 trials demonstrated significant motor ...

The procedure aims to extend the lifespan of blood vessels used during the graft process in the surgery.

An investigational, one-time gene therapy appears to slow the progression of Huntington disease (HD), results of a new phase 1/2 trial suggest. A press release issued by uniQure, the drug's ...

Youth with congenital deafness treated with gene therapy showed better progress on certain hearing measures than counterparts treated with cochlear implantation in a cohort study from China. Gene ...

When she was born, doctors informed Opal Sandy’s parents that their child would never hear. At the age of 11 months, she had not reacted even to the most noise. Two years later, the same child now ...