Usher syndrome

This study tests whether injecting a person's own bone marrow stem cells into or near the eye can help treat various retinal and optic nerve diseases, including age-related macular degeneration, retinitis pigmentosa, and glaucoma. Participants receive stem cell injections via dif…

This study tests an experimental drug called ultevursen for people with retinitis pigmentosa caused by a specific gene mutation (USH2A). The drug is injected into the eye and may help slow vision loss. The trial involves 81 participants, some of whom will receive a sham (fake) in…

This study tests a new treatment called OpCT-001 for people with inherited eye diseases that damage the retina, such as retinitis pigmentosa. The treatment involves injecting special photoreceptor cells, made from stem cells, into the back of the eye to replace lost cells. The tr…

This registry collects information from people with inherited retinal diseases, like retinitis pigmentosa and Stargardt disease. Participants share their symptoms, family history, and genetic test results online. The goal is to help researchers understand these rare diseases and …

This study uses special cameras to take ultra-detailed pictures of the back of the eye in people with retinal diseases like macular degeneration and retinitis pigmentosa. Researchers aim to develop new ways to diagnose and track these conditions by measuring cell density and func…

This study looks at people with retinitis pigmentosa and similar eye diseases who already use a device called OkuStim® that sends mild electrical pulses to the eye. Many patients report temporary improvements in vision right after treatment, but these haven't been measured in a s…

This study follows 60 people with Usher syndrome type 1B, a rare genetic condition causing deafness and progressive vision loss. Researchers will measure how vision changes over time using standard eye tests and new virtual reality tools. No treatment is given; the goal is to bet…