Lisa Kurec never heard of the National Organization for Rare Disorders (NORD) until Wednesday, but after many years of finding no answers for her son’s rare disease, she decided to […]
It took more than seven years to get a genetic diagnosis for our daughter. During that time, doctors were pretty sure she had LCA, although we also heard that maybe […]
Amy Reif still can’t hold back tears when she recounts first hearing that a ground-breaking genetic-therapy treatment targeting her daughter’s LCA-RPE65 gene officially received approval. [...]
This is the first in a series following the progress of Creed Pettit, a 9-year-old Florida third-grader, who is a candidate for the breakthrough gene-therapy drug called LUXTURNA™, approved as [...]
Sofia Sees Hope has launched a Family Connections program designed to help end the isolation that many feel when they receive a rare inherited retinal disease diagnosis. The Family Connections [...]
On Rare Disease Day – Wednesday, February 28 – doctors, researchers, advocates, patients, caregivers, industry representatives and legislators will come together in Connecticut and around the [...]
Ledyard, CT (February 6, 2018) — Sofia Sees Hope, an organization dedicated to finding treatments and cures for blindness caused by inherited retinal diseases (IRDs), is helping to ensure that [...]
Sofia Sees Hope launches International Rare Disease Day February campaign — ‘Get Tested, Get Connected’ — to encourage those diagnosed with rare inherited retinal diseases to receive genetic [...]
Applied Genetic Technologies Corporation (AGTC), a clinical stage biotech company that focuses on rare inherited retinal diseases (IRDs), develops therapies that replace “broken” genes with [...]