The Food and Drug Administration approved Fayuvi (rebisufligene etisparvovec-hopf), made by Ultragenyx Pharmaceutical, on ...

Jessica Rendall is a reporter with experience covering a variety of health and wellness topics, including chronic disease, infectious disease, telemedicine, mental health and health technology. Kelly ...

Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to restore natural hearing for those born with a rare form of deafness, and the ...

For those born with certain types of congenital deafness, the cochlear implant has been a positive and enabling technology. It uses electronics to step in as a replacement for the biological ear that ...

France: A comparative effectiveness study in children with spinal muscular atrophy type 1 (SMA1) has found that gene therapy ...

This voice experience is generated by AI. Learn more. This voice experience is generated by AI. Learn more. Miles, age 2, is one of the first patients at Boston Children's hospital to receive gene ...

NOVATO - Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that the U.S. Food and Drug Administration (FDA) granted standard full approval of FAYUVI (rebisufligene etisparvovec-hopf), also known ...

The special properties of methylcellulose foam could make it a vehicle for bedside genetic engineering, according to a proof-of-principle study from bioengineers at Fred Hutch Cancer Center. In the ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.

Dr. Donald Kohn, a UCLA physician-scientist whose work has helped bring life-saving blood stem cell gene therapies from the ...