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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.
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Leber Congenital Amaurosis (LCA)
LCA is a group of inherited retinal diseases causing blindness or severe vision loss in early childhood.
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Choroideremia is an inherited retinal disease that causes progressive vision loss due to degeneration of the choroid, the retinal pigment epithelium or RPE, and the photoreceptors.
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Often diagnosed in childhood or adolescence, BBS is an inherited disease causing progressive loss of night and peripheral vision from retinitis pigmentosa. BBS can also cause a number of other symptoms and problems including: obesity, extra fingers and toes, kidney disease, and developmental disabilities. Symptoms vary from person to person.
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Best disease is an inherited retinal disease, which causes macular degeneration and loss of central vision, visual acuity, and color perception.
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Achromatopsia is an inherited retinal disease causing loss of visual acuity as well as central and color vision and vision in lighted conditions.