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Displaying 851–860 of 998 results
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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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Stem-Cell Therapy Clinics Remain Inadequately Regulated, Pose Risk to Patients
Research NewsIf a clinic is charging for a stem-cell treatment or procedure for an IRD, it is probably not legit. The expense to the patient is a major red flag.
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ProQR Doses First Participant in Its LCA10 Therapy Clinical Trial
Research NewsThe company plans to report interim, six-month study results in 2018 and 12-month results in 2019.
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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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Beacon Stories
Joe Caruso, founder of FOCUS Wine Foundation, knows life both with and without the ability to see. His first memory is sitting in the waiting room with his mother at the Scheie Eye Institute in Philadelphia.
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The Foundation's Investments Are Filling the Pipeline for Vision-Saving Therapies
Research NewsIn addition to funding promising biotech start-ups, the Foundation Fighting Blindness has played a critical role in developing research talent.
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Heather Presnar’s Story: Living with Stargardt Disease
Beacon StoriesWhen you’re the catcher, the ball always comes to you. Heather Presnar wouldn’t have had it any other way.
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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.
