Supporting the LCA community
We see a cure for blindness
Join Us To Party for Sight!
May – September 7
This summer, we’re inviting you to help shine a light on LCA and other rare inherited retinal diseases (IRDs). Join our community-wide effort to raise awareness and support for Hope in Focus and our mission to advance treatments.
Host a small get-together, share facts about rare diseases, or tell your own story. Big or small, every effort helps spread awareness and brings more attention to our shared journey. Together, we can make a lasting impact that extends far beyond the summer.
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!
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27 known mutations cause LCA—let’s take a closer look at them!![]()
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LCA6 is caused by mutations in the RPGRIP1 gene, which affects about 5-6% of people with LCA. RPGRIP1 is important for a part of the eye’s light-sensing cells called the connecting cilia. Without RPGRIP1, these cells can’t keep their outer segments (the parts that actually detect light) healthy. This leads to early vision loss.![]()
RPGRIP1 was originally found to interact with RPGR, the gene linked to the most common form of X-linked retinitis pigmentosa (RP), and mutations have also been linked to cone–rod dystrophy (CORD13).![]()
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Currently, Odylia Therapeutics is developing a gene therapy called OT-004. This therapy is in late-stage preclinical development, with clinical trials expected to begin soon.![]()
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Read about an adult living with LCA6, Angélica Bretón Morán: loom.ly/L7gZnRU![]()
And the story of Jessi Crawford, a mother whose child has LCA6: loom.ly/Pd59QcY![]()
For more information on Odylia Therapeutics and updates on gene therapy for LCA6: loom.ly/1T994CU![]()
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#LCA #LeberCongenitalAmaurosis #HopeInFocus #LCA6 #RPGRIP1 #BlindCommunity #LowVision #GeneTherapy #Inclusion
Join the Hope in Focus summer fundraiser, Party for Sight!![]()
Host a gathering of any size, from a backyard BBQ to a beach day, and help raise funds and awareness for treatments for rare inherited retinal diseases like Leber congenital amaurosis (LCA). ![]()
Have fun, bring people together, and support a great cause all summer long. We can't wait to see what you came up with!🌊![]()
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Want to start or donate to existing fundraisers? Click this link: loom.ly/ScmJiKk ![]()
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#LCA #lebercongenitalamaurosis #HopeInFocus #partyforsight2025 #awareness #blindcommunity #lowvision #visuallyimpaired #inclusion #fundraiser