Foundation Fighting Blindness - Research and Clinical Trials

Research and Clinical Trials

The Foundation funds research in a number of scientific areas including: genetics, gene therapy, nutrition, stem cells, and pharmaceutical therapies.

After decades of Foundation-funded research, several promising treatments have moved into human clinical trials, including a landmark gene therapy human study for Leber congenital amaurosis, which has enabled more than 40 children and young adults who were virtually blind to read several lines on an eye chart and see in dimly lit settings. This success paved the way for the development of gene therapies now in clinical trials to treat a wide range of other retinal conditions, including Stargardt disease, Usher syndrome, and age-related macular degeneration.

Valproic acid, a drug that is FDA-approved to treat epilepsy, has shown promise for preserving vision in people with certain forms of retinitis pigmentosa. The Foundation has launched a human trial to test this drug and, if effective, move it quickly out to patients who need it.

Foundation-funded researchers are using stem cells derived from a variety of sources, including a person’s own skin, to create healthy retinal cells that can potentially restore vision. Stem cell treatments hold great promise for people with advanced vision loss.

The Foundation is also funding clinical trials of a tiny innovative capsule that is inserted into the eye to slow vision loss from a variety of retinal degenerative diseases.

Read more about this topic:  Foundation Fighting Blindness

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