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,...
Family Stories
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...
Mother of Toddler Living with LCA6 RPGRIP1 Collaborates with Biotech Odylia to Help Advance Gene Therapy Research
After crazy months of looking for answers to questions about her infant’s vision, Melissa Matias learned her baby girl, Dylan,...
Family Excited About New LCA5-Lebercilin Research Program
John Mills says he would crawl over broken glass if it led to a cure for his daughter’s visual impairment...
Opus Genetics Launch Brings Cautious Optimism For RDH12 Families
Allison Galloway feels cautiously excited about a new genetics company prioritizing research into her children’s form of Leber congenital amaurosis...
Big Journey for Little Girl with LCA3
Parvi Gaddam has endured a lot for a little girl genetically diagnosed at 6 months’ old with a form of...
Navigating Life with CRB1
Joseph F. Smith has been legally blind since birth. He lost what little sight he had in his mid-30s, when...
Raising Awareness by Sharing Rare Disease Stories
Had he received a more definitive rare disease diagnosis in 2003, Alan Gunzburg said he might not have lost so...
Michael Kalberer’s Philosophy: Transcend Disability
Born with cerebral palsy (CP), Michael J. Kalberer grew up with a philosophy that has served him well throughout his 43...
Hope is Believing the Future Can Be Better
I must admit, when I learned that my vision was slowly deteriorating, I lost hope. I didn’t know what the...