Family Stories

Theodor Karl Gustav von Leber would be proud. So would Adolphe Franceschetti and Carl-Henry Alström.  Their research from the 19th and 20th centuries laid the foundation for groundbreaking gene [...]

Tell your story. Tell your story again. Then tell it again.   That’s the beginning of advocacy for rare disease.  “You have to be assertive and speak up. You don’t have time to waste!” advocate [...]

Kristen Steele knows a thing or two about telling her story and getting what she needs to be her best. The 22-year-old from Council Bluffs, Iowa, is a licensed massage therapist in Iowa and [...]

Known as a pioneer in gene therapy, Jean Bennett, MD, PhD, surveyed her audience of patients and families living with Leber congenital amaurosis and declared: “YOU are all the pioneers!” Dr. [...]

Advances in genetic sequencing boosted research into rare inherited retinal diseases (IRDs), making a tremendous impact on the number of clinical trials underway for genetic treatments. “There [...]

The long and sometimes uncompromising road to completing the Human Genome Project (HGP) paved the way for today’s surge in genetic therapy, Dr. Katherine A. High said in her presentation at the [...]

The news is out from our 2019 LCA Family Conference  and it’s terrific! Families living with Leber congenital amaurosis (LCA) and other rare inherited retinal diseases (IRDs) learned at Sofia [...]

Superstar geneticists and a host of retinal doctors, researchers, advocates, industry leaders and patients converge in Philadelphia this weekend when Sofia Sees Hope presents its second LCA [...]

Kristen Steele is a trailblazer. The 21-year-old paved a smoother road for those without vision by changing massage-therapy exam protocol, writing policies and procedures, and proving herself as [...]

Heather Le’s anxiety accompanying her daughter’s diagnosis of Leber congenital amaurosis melted into relief when she connected with a mom whose daughter was also born with LCA. And this mom – [...]