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Turns out inherited eye diseases aren’t a sure thing
03 Feb 2026 18:52 GMT
… pigmentosa, inherited macular degeneration, and Leber congenital amaurosis.
The diseases are “monogenic,” which …
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Global Leber Congenital Amaurosis Market 2025-2034: Growth, Share, Segments & Emerging Trends Analysis
13 May 2025 17:14 GMT
… the Growth of the Leber Congenital Amaurosis Market?
The leber congenital amaurosis market is expected to … ;leber-congenital-amaurosis-global-market-report
How Is the Leber Congenital Amaurosis Market Segmented?
The leber congenital amaurosis …
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FDA grants RMAT designation to gene therapy for Leber congenital amaurosis
08 May 2025 01:12 GMT
… -LCA5 for the treatment of Leber congenital amaurosis due to genetic variations in …
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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
21 Feb 2025 11:30 GMT
… the most severe forms of Leber congenital amaurosis 4 (LCA4) owing to genetic …
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MeiraGTx Receives Rare Pediatric Disease Designation from FDA for AAV8-RK-RetGC for the Treatment of Patients with Leber Congenital Amaurosis due to GUCY2D Mutations
22 Jan 2025 13:30 GMT
… the treatment of patients with Leber congenital amaurosis due to GUCY2D mutations (LCA1 … of cases causing autosomal recessive Leber congenital amaurosis type 1 (LCA1) whilst … the most severe forms of Leber congenital amaurosis (LCA) owing to genetic …
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FDA grants rare pediatric disease designation for Leber congenital amaurosis gene therapy
21 Aug 2024 14:09 GMT
… patients with a form of Leber congenital amaurosis, according to a press release … patients with a form of Leber congenital amaurosis, according to a press release …
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Novel Gene Therapy Improves Vision in Children with Leber Congenital Amaurosis
07 May 2024 13:06 GMT
… 17. These participants suffer from Leber Congenital Amaurosis (LCA), a blindness caused by … Mark Pennesi, MD, PhD.
About Leber Congenital Amaurosis
Leber Congenital Amaurosis (LCA) is a severe genetic …
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Opus Genetics completes dosing in first cohort of Leber congenital amaurosis trial
28 Mar 2024 18:13 GMT
… therapy, under investigation for Leber congenital amaurosis, demonstrated signs of biological … for the treatment of Leber congenital amaurosis, according to a press … administration in patients with Leber congenital amaurosis resulting from biallelic mutations …
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Atsena Therapeutics Receives Rare Pediatric Disease Designation from FDA for ATSN-101 Gene Therapy for GUCY2D-associated Leber Congenital Amaurosis (LCA1)
16 Jan 2024 12:30 GMT
… clinical trial in patients with Leber congenital amaurosis caused by biallelic mutations in … ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations … ATSN-101 for GUCY2D-associated Leber congenital amaurosis (LCA1). ATSN-201, which …
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JPM26: Opus Genetics builds momentum in rare retinal gene therapy
16 Jan 2026 14:31 GMT
… advanced program, OPGx-LCA5, targets Leber congenital amaurosis type 5, a rare and …
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