Supporting the LCA and rare retinal disease community
We see 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, Sofia Sees Hope 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 Sofia Sees Hope.
Let’s Get Social!
40+ experts in #ophthalmology & #GeneResearch gathered recently to share research & patient perspectives and identify next steps to advance treatments for #LeberCongenitalAmaurosis caused by variation of the #IQCB1 gene.
https://t.co/Wbsow8XbSh @fightblindness
An incredible lineup of webinars for spring and summer on a host of topics important to the rare inherited retinal disease community. Sign up now - they're free. https://t.co/7thTc6mV6L
We have an incredible lineup of webinars for spring and summer! Don’t miss out on all this important information. https://t.co/7thTc6mV6L
More than 40 leading experts in ophthalmology and gene research gathered for the virtual workshop to share research and patient perspectives and identify the next steps to advance treatment for the patient community in regards to the IQCB1 gene. ![]()
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Sofia Sees Hope and the Foundation Fighting Blindness created the Scientific Advancement Workshop to widen the circle of research awareness, build a framework or platform for sharing knowledge, foster collaboration among stakeholders, identify gaps, and set priorities for action.![]()
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sofiasees.org/give-us-hope-bringing-together-leber-congenital-amaurosis-researchers-and-patients/
sofiasees.org
Sofia Sees Hope and the Foundation Fighting Blindness created the Scientific Advancement Workshop to widen the circle of research awareness.
Great result from ProQR's Phase 1/2 clinical trial of sepofarsen in CEP290 mediated #lebercongenitalamaurosis. Results from the Phase 2/3 Illuminate trial coming next year.![]()
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www.nature.com/articles/s41591-021-01297-7
www.nature.com
Post hoc analyses and follow-up of a patient with Leber congenital amaurosis treated with the antisense oligonucleotide sepofarsen as part of a clinical trial indicates sustained improvement of vision 15 months after receiving a single dose of the drug.