2023 LCA Family Conference: Developing LCA Treatments
Luxturna®, the only approved treatment for one of 27 identified forms of Leber congenital amaurosis (LCA), cost $500 million to develop...
HIF Activities
2023 LCA Family Conference: Living with LCA–Panelists with varied vision share life successes and challenges
Tami Morehouse is grateful for improved vision after undergoing groundbreaking gene therapy treatment at age 44 for LCA2 RPE65, but at times she...
2023 LCA Family Conference: Advocating for Treatments
Here’s what you need to know about advocating for advanced treatments for Leber congenital amaurosis: Get genetically tested, get legislatively connected,...
Hope Rules at 2023 Hope in Focus LCA Family Conference in Indianapolis
Our 2023 LCA Family Conference in Indianapolis offered many reasons to feel hopeful about more retinal disease treatments coming to fruition and...
Ready, Set, Go! Hope in Focus 2023 LCA Family Conference Brings Together Rare Retinal Disease Community in Indianapolis
People living with LCA, family members, retinal doctors, researchers, advocates, and representatives of the biotechnology and pharmaceutical industries converge in...
Let’s Chat About … RARE-X with Karmen Trzupek
Through global data sharing and analysis, the nonprofit RARE-X (the research arm of Global Genes) is working to accelerate treatments for rare...
Connecticut Residents Living with Rare Diseases Voice Concerns to State Lawmakers
One woman called doctor after doctor, only to hear they would not treat her and her two rare pediatric conditions...
Connecticut Rare Disease Advisory Council Begins to Take Shape
The 13-member Connecticut Rare Disease Advisory Council (RDAC) is in the middle of its making, with the announcement of its first six...
Plans in the Works for Connecticut’s New Rare Disease Advisory Council and 2023 Rare Disease Day
Advocates for people living with rare diseases are collaborating with Connecticut Gov. Ned Lamont’s office and leaders of the Connecticut...
Genetic Tests Glean New Diagnoses for People Living with Rare Inherited Retinal Disease
Three people who received diagnoses of Leber congenital amaurosis (LCA) in recent years – but lived most of their lives thinking they...