Family Stories

Successful clinical drug trials are a cornerstone of U.S. Food and Drug Administration approval, such as with LUXTURNA™, a ground-breaking genetic therapy that helps restore vision in Leber [...]

1971 – Just those numbers in white on a black page appeared on the big screen. That’s how Brian Mansfield, PhD., began his presentation to families and patients living with Leber congenital [...]

On October 5-6, 2018, Sofia Sees Hope held its first Family Conference for LCA and IRD families and caregivers. Thank you to all who attended and thank you to our sponsors, who made it all [...]

Sofia Sees Hope is holding its first LCA Family Conference on Saturday in Groton, CT. This event is the culmination of our first five years of work and fulfills our mission to connect LCA [...]

Sofia Sees Hope is holding its first LCA Family Conference on Saturday in Groton, CT. This event is the culmination of our first five years of work and fulfills our mission to connect LCA [...]

Sofia Sees Hope is holding its first LCA Family Conference on Saturday in Groton, CT. This event is the culmination of our first five years of work and fulfills our mission to connect LCA [...]

Dinner in the Dark‘s live auction is like no other! With charming master auctioneer Mike O’Farrell to guide you, our committee has gathered amazing experiences, travel, and more! Get [...]

Transforming laboratory research into real-life therapy for patients is a rare occurrence. But when it does happen, it’s big. Huge, in fact. Ask Dr. Audina M. Berrocal, the pediatric retinal [...]

Gene mutations in the rare inherited retinal disease of Leber congenital amaurosis commonly are referred to by their gene name, such as GUCY2D, RPE65 and CEP290. But sometimes, as LCA patients [...]

By Angélica Bretón Morán I am 22 years old and I have LCA. My name is Angélica Bretón Morán, I am from Mexico, I am 22 years old and I have Leber congenital amaurosis (LCA). Two years ago I [...]