How did Hope in Focus begin? In 2013, after our daughter, Sofia, was diagnosed with Leber congenital amaurosis (LCA) IQCB1/NPHP5, the Foundation for Retinal Research (FRR) asked if my husband [...]
Allison Wolf, whose 9-year-old son Elliot lives with the encroaching blindness of Leber congenital amaurosis (LCA), spoke directly to staff from the country’s regulatory agency to help them [...]
So much more than a typical fundraiser, the 8th Hope in Focus Dinner in the Dark we advertised as “a unique sensory experience,” gave guests just that and much more […]
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 […]
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) [...]
