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Mar 27, 2018
Vision Improvements Reported in Early Stem Cell Trial for Wet AMD
Two patients with advanced wet age-related macular degeneration (AMD) in a Phase I clinical trial demonstrated improved visual acuity sustained for one year after a sheet of retinal pigment epithelial (RPE) cells derived from embryonic stem cells was transplanted under their retinas. Each patient had one eye treated. Vision improvement for one patient was 29 letters or about 6 lines on an eye chart. The other had a gain of 21 letters or about 4 lines.
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Mar 20, 2018
Best Disease Gene Therapy Advances Toward Clinical Trial
Using gene therapy, FFB-funded researchers at the University of Pennsylvania School of Veterinary Medicine (Penn Vet) and Perelman School of Medicine have reversed the disease process in a canine model of Best disease, an inherited form of macular degeneration that can lead to severe vision loss in humans
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Mar 8, 2018
Choroideremia Gene Therapy Moves into Phase 3 Human Study
Nightstar Therapeutics, a retinal-disease, gene-therapy development company in the UK, is advancing its emerging gene therapy for choroideremia into a Phase 3 clinical trial known as STAR. The study will enroll approximately 140 patients at 18 clinical sites in the US, Europe, Canada, and South America.
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Mar 7, 2018
Natural History Study Launches for LCA Caused by Specific Mutation in CEP290
The natural history study will be used to characterize the range of visual function in patients, to evaluate which visual tests may be the most useful for patients with this condition, and to determine the rate of change in visual function over a one-year period.
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Feb 14, 2018
FFB-CRI Investing $7.5 Million in Emerging Therapy for USH2A
The Foundation Fighting Blindness Clinical Research Institute (FFB-CRI) has entered into a partnership with ProQR to develop a retinal therapy for people with Usher syndrome type 2A (USH2A) caused by mutations in exon 13 of the USH2A gene.
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Feb 7, 2018
AGTC Launches XLRP Gene Therapy Clinical Trial at Five Sites in U.S.
Taking place at five locations in the United States, the clinical trial will enroll approximately 15 males with X-linked retinitis pigmentosa caused by mutations in the gene RPGR and will primarily evaluate safety.
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Often diagnosed in childhood or adolescence, Usher syndrome is an inherited disease causing combined hearing loss and vision loss from retinitis pigmentosa. The condition can also cause problems with balance.
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X-linked retinoschisis (XLRS) is an inherited retinal disease causing vision loss due to splitting of the layers of the retina.
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Often diagnosed in childhood or adolescence, Stargardt disease is an inherited form of macular degeneration causing central vision loss. The condition is sometimes referred to as juvenile or early onset macular degeneration.
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Age-Related Macular Degeneration
Age-related macular degeneration, or AMD, is the leading cause of blindness in people 55 and older in developed countries. The condition can lead to significant loss of central vision.