Search
Displaying 321–330 of 526 for “Retinitis pigmentosa”
-
ARVO 2018: Dr. Steve Rose Reports on CRISPR/Cas9 for Inherited Retinal Diseases
Research NewsFFB’s own Dr. Steve Rose, chief scientific officer, reviews our commitment to funding and exploring CRISPR/Cas9 gene editing for inherited retinal disease.
-
ARVO 2018: World's Largest Show and Tell for Innovations in Eye Research
Research NewsMore than 11,000 eye researchers from around the world — including five intrepid members from FFB’s science team — will gather to participate in what is essentially a massive “show and tell” of the latest scientific advancements.
-
Leber Congenital Amaurosis (LCA)
DiseaseLCA is a group of inherited retinal diseases causing blindness or severe vision loss in early childhood.
-
The Assistance Fund Opens New Program for Inherited Retinal Diseases
Research NewsThe Assistance Fund, an independent charitable patient assistance foundation that helps patients and families facing high medical out-of-pocket costs, today launched a new program that offers financial support for individuals with inherited retinal diseases.
-
Clinical Trial to Launch for System Combining Optogenetics and Eyewear
Research NewsThe French biotech GenSight Biologics has received regulatory authorization in the UK to launch the PIONEER Phase 1 \ 2 clinical trial for its GS030 system — a light-sensing gene therapy (optogenetics) coupled with eyewear, which enhances visual stimulation.
-
jCyte Reports Results for Phase 1/2a Clinical Trial for Retinal-Cell Treatment
Research NewsSome participants reported increased light sensitivity, improved color vision, better mobility, and improved reading ability.
-
History Is Made: FDA Approves Spark's Vision-Restoring Gene Therapy
Research NewsKnown as LUXTURNA™ (voretigene neparvovec), the gene therapy restored vision in a clinical trial for people between the ages of 4 and 44 with Leber congenital amaurosis (LCA) caused by mutations in the gene RPE65.
-
Beacon Stories
“I try not to get my hopes up too much, but I never lose hope; I’m determined. I just want to see my son’s face, see him get married, & see my grandchildren. I encourage everyone with a retinal disease to get a genetic test & to never ever give up hope.“
-
Research News
An advisory committee comprised of FDA-selected experts voted unanimously – 16 to 0 – to recommend approval.
-
Research News
Investigational LUXTURNA has the potential to be both the first pharmacologic treatment for an inherited retinal disease (IRD) and the first gene therapy for a genetic disease in the United States
First adeno-associated viral (AAV) vector investigational candidate to reach this stage of FDA review
Investigational LUXTURNA clinical program includes up to four years of efficacy data, with observation ongoing
