Music Brings Together Family Living with LCA6 RPGRIP1
Jessi Crawford fancied the clarinet when she played in her middle school band, while classmate Ted Beaman favored the trombone and guitar. Never did they dream their love of music […]
Hope in Focus Rare Disease Day Statement to Connecticut’s Legislature
Representatives from Hope in Focus joined Connecticut’s Rare Disease Day celebration at the Capitol in Hartford on March 23, 2023. A New England snowstorm postponed the event usually [...]
Second Global RDH12 Scientific Conference Brings Clinical Trials Closer to Reality
The community of people living with Leber congenital amaurosis caused by mutations in the RDH12 gene moved closer to realizing the shared goal of establishing a clinical trial to find […]
Plans in the Works for Connecticut’s New Rare Disease Advisory Council and 2023 Rare Disease Day
Advocates for people living with rare diseases are collaborating with Connecticut Gov. Ned Lamont’s office and leaders of the Connecticut General Assembly’s Public Health Committee to build the [...]
Editas Medicine’s EDIT-101 Clinical Trial Update Brings Mixed News on LCA10 (CEP290) Research
Data from Editas Medicine’s clinical trial on gene-editing therapy showed meaningful outcomes and improvements to treat a form of Leber congenital amaurosis (LCA). The improvements, though, were [...]
Let’s Chat About … Atsena Therapeutics’ LCA Research with Kara Fick and Shannon Boye
Atsena Therapeutics, a clinical-stage gene therapy company focused on reversing and preventing blindness, has an ongoing Phase 1/2 clinical trial evaluating a potential therapy for Leber [...]
A Spectacular Return to Dinner in the Dark 2022
Our 7th Annual Dinner in the Dark came off as an exciting evening of hope and fun, and an opportunity to foster and connect with the mission of Hope in […]
Boy’s vision improves after undergoing Compassionate Use gene therapy in UK for LCA4 (AIPL1)
DJ and Brendan Broadbin came to our Hope in Focus LCA Family Conference with a lot of questions about their little boy’s blindness, and they left with amazing answers leading […]
Paddle for Sight
Celebrate the last day of summer with a paddle on the Mystic River, benefiting Hope in Focus and research to treat blindness! On Wednesday, September 21, the last day of […]
Genetic Tests Glean New Diagnoses for People Living with Rare Inherited Retinal Disease
Three people who received diagnoses of Leber congenital amaurosis (LCA) in recent years – but lived most of their lives thinking they had retinitis pigmentosa (RP) – gave us the […]