gene therapy

The first brain-delivered gene therapy on the market was recently approved in Europe and the U.K. for a rare genetic disorder called AADC deficiency. It causes developmental delays and movement disorders in kids.

Although it’s still unclear what killed the 27-year-old, his death is raising questions about the experiment and the overall prospect of what one ethicist calls designer genetic therapies.

Gene therapy has helped a 9-year-old boy regain enough muscle strength to run. If successful in others, the treatment could change the lives of thousands of children with Duchenne muscular dystrophy.

The Food and Drug Administration approved a new gene therapy for a rare but devastating genetic disorder. The drugmaker says the cost is worth it because it's a one-time treatment that saves lives.

Installment plans and refunds for treatments that don't work are two options getting more attention as ultra-expensive therapies become more common. The financial strains will only grow.

U.S. health officials are eliminating special regulations for gene therapy experiments, saying that what was once exotic science is quickly becoming an…

A first-of-its kind genetic treatment for blindness will cost $850,000 per patient, making it one of the most expensive medicines in the world and raising…

The Food and Drug Administration approved Luxturna, a genetically modified virus that restores by ferrying a healthy gene into the eyes of patients born with a genetic disease that impairs sight.

After decades of hope and disappointment, doctors have now been able to treat several different types of genetic conditions by giving each patient a healthy version of their defective gene.

Researchers at the University of Florida have developed a new therapy that they hope could lead to a cure for multiple sclerosis.The process uses a gene…