FDA advisers endorse gene therapy to treat form of blindness
SILVER SPRING, Md. -- A panel of U.S. health advisers has endorsed an experimental approach to treating inherited blindness, setting the stage for the likely approval of an innovative new genetic medicine.
A panel of experts to the Food and Drug Administration voted unanimously in favor of Spark Therapeutics' injectable therapy, which aims to improve vision in patients with a rare mutation that gradually destroys normal vision. The vote amounts to a recommendation to approve the therapy.
According to Spark Therapeutics' website, inherited retinal diseases are a group of rare blinding conditions caused by one of more than 220 genes. Some living with these diseases experience a gradual , while others may be born without the ability to see or lose their vision in infancy or early childhood.
Genetic testing is the only way to verify the exact gene mutation that is the underlying cause of the disease.
On Thursday, children, parents, doctors and scientists spoke to the FDA panel about what it's like to lack and then gain one of our most primal senses.
Cole Carper, an 11-year-old boy who got the therapy when he was 8, describes how sight changed what he knew of the world. When he returned to his home in Little Rock, Arkansas, after treatment, "I looked up and said, 'What are those light things?' And my mom said, 'Those are stars.'"
Continue reading at CBSNews.com.