Blog

One woman called doctor after doctor, only to hear they would not treat her and her two rare pediatric conditions because she turned 18 and no longer qualified for help. […]

The 13-member Connecticut Rare Disease Advisory Council (RDAC) is in the middle of its making, with the announcement of its first six members. The council will give patients, families, [...]

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 […]

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 […]

As general manager and vice president of Cardinal Honda in Groton, Conn., Kim Cardinal Piscatelli donates time and money to charities. But when she heard about Hope in Focus and […]

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 [...]

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 [...]

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 [...]

We’re excited to announce what the Dinner in the Dark menu was for 2022! If you’ve never been to our Dinner in the Dark event, this is our primary fundraiser […]

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 […]