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Displaying 341–350 of 543 for “Retinitis pigmentosa”
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Leber Congenital Amaurosis (LCA)
DiseaseLCA is a group of inherited retinal diseases causing blindness or severe vision loss in early childhood.
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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.
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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.
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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.
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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.
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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.“
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Research News
An advisory committee comprised of FDA-selected experts voted unanimously – 16 to 0 – to recommend approval.
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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
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MeiraGTx Treats First Patient in XLRP Gene-Therapy Trial
Research NewsThe MeiraGTx gene therapy involves injection of healthy copies of RPGR underneath the retina. The RPGR copies are contained in a human-engineered virus — known as an adeno-associated virus or AAV — designed to readily penetrate retinal cells to deliver the therapeutic genetic cargo.
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Research News
The partnership will help companies and researchers quickly obtain and implement high-quality vectors for their retinal gene-therapy development efforts.
