Celebrating Connecticut Rare Disease Day 2022, Hope in Focus presented remarks Friday, February 25, 2022, to the Connecticut State Legislature on behalf of those living with Leber congenital [...]
Rare Disease Day 2022 kicked off today as people gathered across the country, including us here at Hope in Focus, to raise awareness and generate research for treatments and cures […]
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 [...]
The big news just keeps coming: fresh off the rebranding from Sofia Sees Hope to Hope in Focus, we are pleased to announce a pledge to raise $300,000 by 2025 […]
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 […]