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David Schulz has been appointed to our Board of Directors. He began his career at Pfizer Central Research as a Research Scientist in 1989 and was promoted to Executive Director […]

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 [...]

Working with preclinical data from multiple Leber congenital amaurosis (LCA) studies at the same time, Opus Genetics hopes to advance research into gene therapy for several forms of LCA at a [...]

Children living with visual impairment become more independent and empowered when parents set high expectations for their kids and challenge them every day. Just ask Beth Borysewicz. In her role [...]

After crazy months of looking for answers to questions about her infant’s vision, Melissa Matias learned her baby girl, Dylan, had a form of Leber congenital amaurosis known as LCA6 caused […]

ProQR Therapeutics’ clinical trials of sepofarsen to treat a form of Leber congenital amaurosis (LCA) caused by a mutation in the CEP290 gene did not meet its primary endpoint of […]

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 [...]