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!
We join with dozens of organizations representing the 30M Americans with rare diseases to support the Rare Disease Advancement, Research, and Education Act of 2018 (H.R.5115) “RARE Act of 2018.” https://t.co/loeMXGDKNW #raredisease
We join with dozens of organizations representing the 30M Americans with rare diseases to support the Rare Disease Advancement, Research, and Education Act of 2018 (H.R.5115) “RARE Act of 2018.” https://t.co/loeMXGDKNW #raredisease
— Sofia Sees Hope (@SofiaSeesHope) April 25, 2018
It’s @DNAday - and we want to remind you how important it is to #KnowYourGene when you have a rare inherited retinal disease ( #IRD)! Knowing your gene can connect you to important research and to others w/ your disease. https://t.co/afdVCXmnc5
It’s @DNAday - and we want to remind you how important it is to #KnowYourGene when you have a rare inherited retinal disease (#IRD)! Knowing your gene can connect you to important research and to others w/ your disease. https://t.co/afdVCXmnc5
— Sofia Sees Hope (@SofiaSeesHope) April 25, 2018
The @US_FDA granted @MeiraGTx Fast Track designation for its experimental #genetherapy AAV-RPGR for treatment of X-linked #retinitispigmentosa due to defects in the retinitis pigmentosa #RPGR gene, an inherited eye disease that causes blindness. https://t.co/UFfENkq7Xf #IRD
The @US_FDA granted @MeiraGTx Fast Track designation for its experimental #genetherapy AAV-RPGR for treatment of X-linked #retinitispigmentosa due to defects in the retinitis pigmentosa #RPGR gene, an inherited eye disease that causes blindness. https://t.co/UFfENkq7Xf #IRD
— Sofia Sees Hope (@SofiaSeesHope) April 24, 2018
Wednesday marked a milestone after Creed’s surgery, reaching what doctors call the 30-day peak, meaning 30 days after surgery on Creed’s right eye, his vision will have improved as much as it is going to from the treatment. https://t.co/LpORwgIQJ4 #Luxturna https://t.co/1hpKK0GCpd
Wednesday marked a milestone after Creed’s surgery, reaching what doctors call the 30-day peak, meaning 30 days after surgery on Creed’s right eye, his vision will have improved as much as it is going to from the treatment. https://t.co/LpORwgIQJ4 #Luxturna pic.twitter.com/1hpKK0GCpd
— Sofia Sees Hope (@SofiaSeesHope) April 20, 2018
Today is International Guide Dog Day! We're celebrating the hard work guide dogs put in to give independence to their owners by giving a shout out to the largest Guide Dog school in North America, Guide Dogs for the Blind! Learn more about their mission to serve people who are blind or low vision here: www.guidedogs.com/
Image Description: A yellow Lab guide dog is lying down, wearing a guide dog harness.
Today is "National DNA Day," which celebrates the completion of the Human Genome Project 15 years ago. And here's a news story that once again underscores why it is so important to #KnowYourGene if you have a rare inherited retinal disease.
When you know your gene, and you make sure you are listed in Foundation Fighting Blindness's My Retina Tracker, then you make yourself visible to researchers who are working on cures ...
When you know your gene, you can find others in the rare disease universe who have the same genetic mutation. There is a lot to be said for having someone to talk to, a support group, folks around you who understand what you are going through.
It's why in 2018, SSH's education and awareness campaign is "#KnowYourGene: Get Tested, Get Connected."
Meira GTx
FDA Grants Fast Track Designation to MeiraGTx Gene Therapy for Rare Eye Disease
globalgenes.org
Rare Daily Staff The U.S. Food and Drug Administration granted MeiraGTx Fast Track designation for its experimental gene therapy AAV-RPGR for the treatment of X-linked retinitis pigmentosa due to d…