Family Stories

Tami Morehouse is grateful for improved vision after undergoing groundbreaking gene therapy treatment at age 44 for LCA2 RPE65, but at times she still is sad and disappointed at the […]

Here’s what you need to know about advocating for advanced treatments for Leber congenital amaurosis: Get genetically tested, get legislatively connected, and get enrolled in a patient registry. [...]

Our 2023 LCA Family Conference in Indianapolis offered many reasons to feel hopeful about more retinal disease treatments coming to fruition and in less time than the dozen or more […]

People living with LCA, family members, retinal doctors, researchers, advocates, and representatives of the biotechnology and pharmaceutical industries converge in the racing capital of the world [...]

Five-year-old Noah Johnson lives in a special place where he can see rockets shoot into the air at night. “I can see,” he yells to his mom, Stephanie. “I can […]

Through global data sharing and analysis, the nonprofit RARE-X (the research arm of Global Genes) is working to accelerate treatments for rare diseases, including Leber congenital amaurosis (LCA) [...]

Desirae Potts breaks into tears when she recalls the first time a doctor said her infant son James had a disease she’d never heard of – Leber congenital amaurosis, known […]

One woman called doctor after doctor, only to hear they would not treat her and her two rare pediatric conditions because she turned 18 and no longer qualified for help. […]

The 13-member Connecticut Rare Disease Advisory Council (RDAC) is in the middle of its making, with the announcement of its first six members. The council will give patients, families, [...]

Jessi Crawford fancied the clarinet when she played in her middle school band, while classmate Ted Beaman favored the trombone and guitar. Never did they dream their love of music […]