Inherited retinal dystrophy

This study tested a single injection of a gene therapy called GT005 in 98 people with geographic atrophy, an advanced form of dry age-related macular degeneration that causes vision loss. The goal was to see if the treatment could slow the growth of damaged areas in the eye and c…

This study tested a single injection of GT005, a gene therapy, in 255 people with geographic atrophy from dry age-related macular degeneration. The goal was to see if it could slow the growth of blind spots in the eye. The trial was terminated early, so we have less data than hop…

This early-stage trial tested a gene therapy called GT005 for dry age-related macular degeneration (AMD), a leading cause of vision loss. The therapy delivers a working gene to the retina via a single injection. The study enrolled 56 people and focused on safety and dosing, but w…

This study tested a gene therapy for retinitis pigmentosa, an inherited eye disease that causes gradual vision loss. The treatment involved injecting a corrected gene under the retina in one eye. The trial included 19 people aged 13 and older with a specific genetic defect. The m…

This study tested whether using a virtual reality headset for one-hour sessions could help regenerate damaged optic nerves and improve vision in people with glaucoma or other retinal diseases. The idea came from promising results in rodents. However, the trial was terminated earl…

This Phase 2 study tested an oral drug called danicopan in 365 people with geographic atrophy, an advanced form of dry age-related macular degeneration that causes blind spots. Participants took different doses of danicopan or a placebo for up to 104 weeks to see if it could slow…

This study looked at whether eating corn rich in zeaxanthin (a natural pigment) could increase the density of the macular pigment in the eye, which may help prevent age-related macular degeneration (AMD), a leading cause of blindness. Nine healthy volunteers aged 20-35 ate a dail…

This study looked at people with cone disorders, a type of inherited eye disease that affects color vision and sharp sight. Researchers analyzed genetic changes of unknown significance to see if they cause disease. The goal was to improve genetic diagnosis, not to test a treatmen…

This study tested a new, non-invasive eye imaging method called hyperspectral imaging. It takes pictures of the back of the eye using many different colors of light to find details not visible with standard cameras. About 679 adults with healthy eyes or eye diseases like diabetic…

This study followed 7 people with Mucolipidosis Type IV (MLIV) to learn how the disease naturally progresses. Researchers used tests like neuropsychological exams, blood and urine tests, and brain MRIs to find better ways to measure the disease. The goal was to improve future cli…

This study looked at over 100 people with inherited retinal dystrophies, a group of rare eye diseases that can cause vision loss. Researchers collected genetic and eye exam data to find links between specific gene mutations and symptoms. The goal was to better understand these di…