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Displaying 411–420 of 537 news results
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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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A Retinal Research Nonprofit Paves the Way for Commercializing Gene Therapies
In the PressAN EMERGING, vision-restoring gene therapy for a devastating retinal disease is poised for Food and Drug Administration (FDA) approval. If it gets the regulatory nod, it will be the first gene therapy to receive FDA approval for the eye or an inherited condition.
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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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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
