FFB Written Articles » Other Retinal Diseases
Oral Compound Improves Vision in Three People with LCA
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Three people with severe vision loss from Leber congenital amaurosis (LCA) have experienced improvements in vision after receiving an oral compound in an early stage clinical trial. QLT Inc., the developer of the compound, presented interim results of their clinical study on May 3 at the 2010 annual meeting of the Association for Research in Vision and Ophthalmology in Ft. Lauderdale. The individuals in the study — ages 10, 12 and 38 — have severe, early onset vision loss from LCA caused by variations in the gene LRAT. In people with this form of LCA, 11-cis-retinal, a biochemical necessary for vision, is not produced. QLT’s treatment is a synthetic replacement for 11-cis-retinal. The study participants experienced improvements in one or more of the following: visual acuity, peripheral vision, and retinal sensitivity. The improvements were observed after participants received the treatment for seven days. Effects of the treatment lasted as long as four months QLT says the study is ongoing, and that they plan to enroll a total of eight participants, primarily children ages 5 to 14. In 2001, Foundation-funded researchers from the University of Michigan discovered that variations in LRAT were a cause of LCA. That finding, and the identification of the missing biochemical 11-cis-retinal in affected individuals, enabled QLT and their researchers to identify potential treatments for the condition. |
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