Family Stories

It’s you. It’s all about you. The journey from identifying a rare disease, to conducting studies, to approving a treatment, is long – but it always starts with the patient, and the information [...]

Two little girls – one on the East Coast and one on the West Coast – ring in this New Year with something they’ve never had before – their vision. Eight-year-old Hannah Reif of Maple Glen, PA, [...]

The lack of information on rare diseases can create difficulty in developing drugs to treat them. To help, it is important to study the natural history of rare diseases. Compared with common [...]

Mikayla Larson, a 30-year-old mother living with a rare inherited retinal disease (IRD) called Leber congenital amaurosis (LCA) wants to reassure children living with the same disease that [...]

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 [...]