Giving Tuesday: Help Provide Access to Free Genetic Testing
When our daughter Sofia was 2, we knew something was wrong with her vision. By the time she was 5,...
HIF Activities
Retinal Disease Gene Therapy Breakthroughs Trace Their Roots to 19th Century Research
Theodor Karl Gustav von Leber would be proud. So would Adolphe Franceschetti and Carl-Henry Alström. Their research from the 19th and 20th centuries laid the foundation...
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...
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...
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....
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...
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, Hope in Focus...
The Patient Voice: A Critical Piece of the Rare Disease Advocacy Puzzle
Two people deeply involved in patient advocacy and public policy recently urged members of the rare disease community to use...
Rare Disease Advocacy: ‘Everything’s connected to everything’
Long before Grey’s Anatomy captured television audiences, the 1980s medical drama, Quincy, M.E., helped propel passage of Congressional legislation that fostered the development of...
CT Rare Action Network Advocacy Workshop May 3
Learn more about rare disease patient advocacy and connect with the National Organization for Rare Disorders (NORD) about current legislative action on...