Archives

Luxturna®, the only approved treatment for one of 27 identified forms of Leber congenital amaurosis (LCA), cost $500 million to develop and took more than 12 years to come to […]

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 […]

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. […]

Hope in Focus is pleased to announce our Director of Outreach and Development Courtney Coates will be attending the largest meeting of eye and vision researchers in the world. The […]

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, [...]