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A proposed Congressional Act designed to help researchers launch clinical trials for emerging treatments gives hope for getting more treatments across the finish line for people living with a [...]

ProQR Therapeutics’ Founder and Chief Executive Officer shared exciting news of the deep pipeline of RNA therapies in development to treat Leber congenital amaurosis (LCA) and other [...]

One of Britain’s top ophthalmologists delivered promising research news on several forms of Leber congenital amaurosis, including LCA13 (RDH12), LCA1 (GUCY2D), LCA2 (RPE65), and LCA4 (AIPL1). [...]

John Mills says he would crawl over broken glass if it led to a cure for his daughter’s visual impairment caused by one of the rarest of rare inherited retinal […]

Allison Galloway feels cautiously excited about a new genetics company prioritizing research into her children’s form of Leber congenital amaurosis known as LCA13 (RDH12).  “I would say I’m [...]

Parvi Gaddam has endured a lot for a little girl genetically diagnosed at 6 months’ old with a form of Leber congenital amaurosis called LCA3 (SPATA7). Like most parents discovering […]

For the first time, early research data shows that a gene editing technique called CRISPR improved vision in people living with a form of Leber congenital amaurosis (LCA), according to […]

Sofia Sees Hope this month unveiled a new name and logo — Hope in Focus — as the organization sets its course for the next decade of work to benefit […]

Why did we change our name? It’s simple: we’ve grown and matured as an organization. And we’re looking ahead to how we can best position this organization for the future. […]

We hear a lot these days about the necessity of the patient voice in developing treatments, especially for people living with rare disease, such as Leber congenital amaurosis (LCA) or […]