The A to Z on LCA and IRDs
Inherited retinal diseases, or IRDs, are rare eye disorders caused by an inherited gene mutation. Depending on the particular IRD, people can gradually lose their vision or they can be […]
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To Test or Not To Test: 5 reasons we think you can and should get a genetic diagnosis for your IRD
Hey LCA or IRD family, does any of this sound familiar? You or your child has received a clinical diagnosis of LCA (leber congenital amaurosis), RP (retinitis pigmentosa), cone-rod dystrophy, [...]
2nd ‘Blindfold Breakfast’ Challenge Launched for World Rare Disease Day
(Feb. 23, 2017) — On Feb. 28, World Rare Disease Day, Sofia Sees Hope launches its second annual Blindfold Breakfast Challenge, a social media campaign designed to bring awareness to […]
ProQR Announces Updates and Priorities After Illuminate Trial Analyses
ProQR Therapeutics completed an in-depth strategic review to prioritize its objectives toward advancing RNA therapies, following news that its Illuminate Phase 2/3 clinical trial of sepofarsen in [...]
Post-Surgery: Beginning to See the Light
This is the fourth in a series following the progress of Creed Pettit, a 9-year-old Florida third-grader, who completed treatment in March with the breakthrough gene-therapy drug called [...]