Rare Disease Advocacy: There’s Power In Numbers
Tell your story. Tell your story again. Then tell it again. That’s the beginning of advocacy for rare disease. “You have to be assertive and speak up. You don’t have time to waste!” advocate [...]
Kristen Steele: You’ve Gotta Fight for Your Right …
Kristen Steele knows a thing or two about telling her story and getting what she needs to be her best. The 22-year-old from Council Bluffs, Iowa, is a licensed massage therapist in Iowa and [...]
Dr. Jean Bennett: ‘Seeing the Light with Retinal Gene Therapy’
Known as a pioneer in gene therapy, Jean Bennett, MD, PhD, surveyed her audience of patients and families living with Leber congenital amaurosis and declared: “YOU are all the pioneers!” Dr. [...]
Explosive Growth Seen in Field of Rare Inherited Retinal Disease Research
Advances in genetic sequencing boosted research into rare inherited retinal diseases (IRDs), making a tremendous impact on the number of clinical trials underway for genetic treatments. “There [...]
Human Genome Project: Critical to Modern Gene Therapy Success
The long and sometimes uncompromising road to completing the Human Genome Project (HGP) paved the way for today’s surge in genetic therapy, Dr. Katherine A. High said in her presentation at the [...]
LCA Family Conference 2019: A Lot to Unpack!
The news is out from our 2019 LCA Family Conference and it’s terrific! Families living with Leber congenital amaurosis (LCA) and other rare inherited retinal diseases (IRDs) learned at Sofia [...]
LCA Family Conference 2019 Brings Together Rare Retinal Disease Community
Superstar geneticists and a host of retinal doctors, researchers, advocates, industry leaders and patients converge in Philadelphia this weekend when Sofia Sees Hope presents its second LCA [...]
When Self-Advocacy Creates Change That Benefits Everyone
Kristen Steele is a trailblazer. The 21-year-old paved a smoother road for those without vision by changing massage-therapy exam protocol, writing policies and procedures, and proving herself as [...]
LCA Families Finding Strength with Each Other
Heather Le’s anxiety accompanying her daughter’s diagnosis of Leber congenital amaurosis melted into relief when she connected with a mom whose daughter was also born with LCA. And this mom – [...]
Diagnosis to Treatment: Pioneering LCA Patient Eases the Journey
As a global advocacy organization dedicated to helping those affected by blindness caused by rare inherited retinal disease, Sofia Sees Hope connects families with Leber congenital amaurosis [...]