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!
. @ProQR Doses First Patient in Phase 2/3 ILLUMINATE Trial of Sepofarsen for LCA10.
https://t.co/pOkjrWzb50 #LCA #IRD #weseeacure
Another milestone reached by @AGTC_ in gene therapy. It's not for #LCA, but every step forward for one is a step forward for all. https://t.co/cQIXvdgZLy #CNGB3trial #raredisease
A few tickets left for the evening.
A couple tickets left for the rare champagne tasting. We'd love to share this Rare Opportunity with you.
https://t.co/3OvPovl93P @bsb_savoy @PinePointSchool
Important news for those in our community with CEP290.
"ProQR Therapeutics N.V. today announced the first patient dosed in the Phase 2/3 ILLUMINATE clinical trial for sepofarsen (formerly named QR-110) in patients with
Leber’s Congenital Amaurosis 10 (LCA10). Data from the study are expected at year-end 2020."
ProQR Doses First Patient in Phase 2/3 ILLUMINATE Trial of Sepofarsen for LCA10
globenewswire.com
LEIDEN, The Netherlands and CAMBRIDGE, Mass., April 15, 2019 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced the first patient dose...This is fantastic.
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