This approval was followed by Takeda Pharmaceuticals’ Alofisel (darvadstrocel), which received approval from the European Medicines Agency in 2018 and from Japan’s Pharmaceuticals and Medical Devices ...

Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to ...

Gene therapy introduces genes to a patient’s body to guide encoded proteins to work favorably against targeted diseases. In the late 1980s, it entered the spotlight as the next generation in ...

Ten out of 11 children with a form of congenital deafness who were treated with an experimental gene therapy developed by Regeneron have seen notable improvements in hearing, according to new data ...

Bharat Biotech aims to democratize gene therapies, traditionally considered prohibitively expensive and available primarily ...

Gene therapy has allowed several children born with inherited deafness to hear. A small study published Wednesday documents significantly restored hearing in five of six kids treated in China.

Stem cell therapy has emerged as a groundbreaking treatment option for corneal injuries, offering hope to individuals suffering from severe damage to the cornea.Cornea is the transparent, dome-shaped ...

Learning from examples like congenital heart disease and cystic fibrosis can help health systems and clinicians prepare to care for an influx of patients with neuromuscular diseases as they reach ...