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0 0.14854517611026 0.0735068912710566 0.110260336906585 0.14854517611026 0.162327718223583 0.188361408882083 0.188361408882083
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MeiraGTx Announces The Lancet Publication of Data Demonstrating the Efficacy of rAAV8.hRKp.AIPL1 for the Treatment of Leber Congenital Amaurosis 4 (LCA4) Retinal Dystrophy

MeiraGTx Holdings plc - Ordinary Shares (MGTX) 
Company Research Source: GlobeNewswire
As disclosed in the paper, 4 out of 4 young children with the AIPL1-related retinal dystrophy, LCA4, benefited substantially from unilateral subretinal administration of rAAV8.hRKp.AIPL1 with improved visual acuity, functional vision, and protection against progressive retinal degenerationFollowing the strong safety and substantial efficacy demonstrated in this first cohort of 4 children treated unilaterally, a further 7 children have now been treated bilaterally and all showed substantial benefit from treatment with rAAV8.hRKp.AIPL1Meaningful responses have been observed in 11 out of 11 LCA4 children treated to date with rAAV8.hRKp.AIPL1All 11 children treated with rAAV8.hRKp.AIPL1 between 1 and 4 years old were legally blind at birth, and all gained visual acuity after 4 or more weeks following a single one time delivery of rAAV8.hRKp.AIPL1Webcast today at 8:00 a.m. ET. Register here LONDON and NEW YORK, Feb. 21, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a ve Show less Read more
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