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Promising gene therapy research – characterized as having a good potential to restore vision – is underway to help visual impairment caused by a form of Leber congenital amaurosis (LCA) [...]

Researchers and regulators did not miss a single step in the fast-tracked federal process of developing successful COVID-19 vaccines. And the same goes for fast-tracking gene therapies for rare [...]

Had he received a more definitive rare disease diagnosis in 2003, Alan Gunzburg said he might not have lost so much vision and still might be able to drive. In 2016 – 13 years after his initial [...]

15 states with an established Rare Disease Advisory Council: Alabama, Kentucky, Illinois, Massachusetts, Minnesota, Missouri, Nevada, New Hampshire, New York, North Carolina, Ohio, Pennsylvania, [...]

Fifteen states done, 35 to go. Fifteen states have established a Rare Disease Advisory Council (RDAC) to give the rare disease community, including those living with Leber congenital amaurosis [...]

In the debut of Sofia Sees Hope’s ‘Let’s Chat About …’ monthly webinar series, Ben Shaberman of the Foundation Fighting Blindness, provided his Zoom audience with a plethora of information [...]

ProQR Therapeutics reached an important milestone by completing enrollment in the next pivotal phase of clinical trials of sepofarsen, a developing RNA therapy for treating LCA10, a form of Leber [...]

Born with cerebral palsy (CP), Michael J. Kalberer grew up with a philosophy that has served him well throughout his 43 years. “My parents raised me as an individual with a disability, not a [...]

Danielle Senick of Norwich, Conn., reached out to Sofia Sees Hope more than three years ago when she needed answers about her deteriorating vision. Doctors diagnosed Danielle at age 6 months with [...]

Building on her son’s love of music, singing, moving, and reading, Laura Steinbusch created a multilingual children’s songbook called Lux+Louise to help youngsters learn music by braille. With [...]