FORMERLY KNOWN AS SOFIA SEES HOPE
SEEING A CURE FOR BLINDNESS
What is Leber Congenital Amaurosis (LCA)?
LCA is a rare inherited retinal disease characterized by severe vision loss at birth. While some children are born with little or no vision, others may have significant vision loss in the first few years of life, stable vision for a period of time, and then eventually complete vision loss as the retina deteriorates into total blindness.
What We Do
Founded in 2014 as Sofia Sees Hope, Hope in Focus is a nonprofit 501(c)3 patient advocacy organization dedicated to generating awareness, raising funds for research, and providing support, education and outreach to the LCA and rare inherited retinal disease community.
Thank you to these organizations
for their ongoing commitment to Hope in Focus.
Let’s Get Social!
Exciting news! The Phase 1/2, open-label, dose-escalation trial will evaluate the subretinal delivery of OPGx-001 in nine adult patients with LCA5.
opusgtx.com
OPGx-001 is Opus’ first program to enter clinical evaluation and is designed to address vision loss due to mutations in the LCA5 gene, which causes one of the most severe forms of early-onset blindi...
We were grateful to hear the latest Eye on the Cure Podcast episode which talked about about retinal disease gene therapies including positive results from a gene therapy clinical trial for people with Leber Congenital Amaurosis 1 (GUCY2D mutations). ![]()
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Listen to this episode at: www.FightingBlindness.org/news/eyecurepodcast-305
www.fightingblindness.org
The Eye on the Cure Podcast from the Foundation Fighting Blindness provides science information, news, and insights from the world of vision and retinal diseases. The podcasts are hosted by Ben Shaber...