“If someone told me that having access to perfect vision tomorrow meant not having impaired vision for my entire life, I would not change the past. While my visual impairment has created many challenges, it has shaped who I am and influenced me in many positive ways. I’ve become more resourceful, compassionate, creative, and resilient.” —Jack McCormick Jack McCormick lives a story of hope, tenacity, and measured success while embracing an unknown visual future. When Jack was two, a diagnosis of cone-rod dystrophy confirmed that he had impaired vision. As a teenager, genetic testing revealed th ..read more

How did Hope in Focus begin? In 2013, after our daughter, Sofia, was diagnosed with Leber congenital amaurosis (LCA) IQCB1/NPHP5, the Foundation for Retinal Research (FRR) asked if my husband and I would be willing to raise funds for treatments for her genetic mutation. The FRR was started in 1998 by David and Betsy Brint, whose son had LCA. Little was known then about the disease, and there wasn’t an LCA community when they began. The Brints faithfully stewarded the research and a handful of LCA families to grow that organization into a real community. So, we said yes to the FRR’s request and ..read more

Allison Wolf, whose 9-year-old son Elliot lives with the encroaching blindness of Leber congenital amaurosis (LCA), spoke directly to staff from the country’s regulatory agency to help them understand the dire need for treatments for the rare inherited retinal disease. Elliot Wolf “Elliot’s eyes are dying, Allison said. “They are dying, and we have nothing to help slow down the progressive loss of his eyesight,” she told the Food and Drug Administration (FDA). “What would a timely and effective treatment mean? The whole world,” the mother of four said. Allison and five others affected by LCA ..read more

So much more than a typical fundraiser, the 8th Hope in Focus Dinner in the Dark we advertised as “a unique sensory experience,” gave guests just that and much more Saturday, Oct. 14, 2023, at the Mystic Marriott Hotel & Spa in Groton, Connecticut. Dinner in the Dark afforded the opportunity for guests to experience their dinner in a different way – wearing blindfolds. For a short time it offers a small taste of what it might be like for someone dealing with a rare retinal disease. This key fundraising event supports our mission of “Seeing a cure for blindness,” as we advocate for people l ..read more

Luxturna®, the only approved treatment for one of 27 identified forms of Leber congenital amaurosis (LCA), cost $500 million to develop and took more than 12 years to come to market. With such an enormous investment in time and money, it would make sense to use that same platform for developing new treatments to improve vision or halt progression of blindness. But that’s just not how it works, said Chad R. Jackson, PhD, Senior Director of the Foundation Fighting Blindness Preclinical Transitional Acceleration Research Program. Every individual clinical study must complete a set of rigorou ..read more

Hope in Focus will host its signature fundraising event of the year – Dinner in the Dark – on Saturday, Oct. 14, 2023, at the Mystic Marriott Hotel & Spa in Groton. Dinner in the Dark is a highly anticipated event for the Ledyard-based 501(c)(3) nonprofit organization. Guests can expect a unique sensory and culinary experience, while wearing a blindfold and enjoying a gourmet meal. The menu is secret, known only to guests who can read a Braille-scripted menu. Hope in Focus provides information and support globally to individuals and families affected by Leber congenital amaurosis (LCA), a ..read more

Tami Morehouse is grateful for improved vision after undergoing groundbreaking gene therapy treatment at age 44 for LCA2 RPE65, but at times she still is sad and disappointed at the difficulties presented to her as a person with some functional vision, but who cannot see well. Mohamed Farid found life living with LCA5 more challenging as a child than as an adult, especially before screen readers and other assistive technology. The young professional also remembers growing up and his grandmother spending a lot of time convincing him he couldn’t be a pilot. Mirielle St. Arnaud, a junior in high ..read more

People living with LCA, family members, retinal doctors, researchers, advocates, and representatives of the biotechnology and pharmaceutical industries converge in the racing capital of the world this weekend, when Hope in Focus presents its third LCA Family Conference. A global advocacy organization helping those affected by blindness, Hope in Focus connects families living with Leber congenital amaurosis (LCA) and other rare inherited retinal diseases (IRDs) through its Family Connections program and through events such as this conference at the Omni Severin Hotel in downtown Indianapolis f ..read more

Five-year-old Noah Johnson lives in a special place where he can see rockets shoot into the air at night. “I can see,” he yells to his mom, Stephanie. “I can see the rocket!” One of the big joys of living in Melbourne on Florida’s Space Coast is witnessing dramatic liftoffs from nearby Cape Canaveral, especially when it’s dark. “It’s so heartwarming because he can see what everyone else can see,” his mom said. Noah also can see colors, faces, and at night, he can see the moon. During the day, he can hear the booming roar of the magnificent engines, but unlike in darkness, he can’t see the spac ..read more

Through global data sharing and analysis, the nonprofit RARE-X (the research arm of Global Genes) is working to accelerate treatments for rare diseases, including Leber congenital amaurosis (LCA) and other rare inherited retinal diseases (IRDs). Hope in Focus featured Karmen Trzupek, RARE-X’s Senior Director of Scientific Programs, in its webinar episode “Let’s Chat About…RARE-X.’ Our Director of Outreach and Development Courtney Coates discussed with Karmen RARE-X’s mission and goals, and its recent merger with Global Genes. The March 7, 2023, session can be viewed here. “Let’s Chat About…” i ..read more