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Retinal organoids provide a powerful tool to diagnose and study Leber congenital amaurosis
04 Dec 2025 02:53 GMT
Leber Congenital Amaurosis (LCA) is an inherited retinal …
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Jael is Deaf and blind. Here's how she goes about her day
06 Dec 2025 20:24 GMT
… work.
Jael was born with Leber congenital amaurosis, a rare inherited eye disease …
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<![CDATA[Positive pediatric data emerge from the OPGx-LCA5 phase 1/2 trial of Leber congenital amaurosis type 5]]>
02 Oct 2025 12:33 GMT
… , an investigational gene therapy for Leber congenital amaurosis type 5 (LCA5), an ultra … to address a form of Leber congenital amaurosis that results from biallelic mutations …
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Sepul Bio, an innovative business unit of Théa, Doses First Participant in Phase 3 HYPERION clinical trial of sepofarsen for CEP290-associated Leber Congenital Amaurosis Type 10
01 Oct 2025 16:59 GMT
… , in individuals with CEP290- associated Leber Congenital Amaurosis Type 10 (LCA10).
“There are …
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Opus Genetics Reports Positive Pediatric Data from OPGx-LCA5 Phase 1/2 Trial in Leber Congenital Amaurosis Type 5 (LCA5)
30 Sep 2025 11:00 GMT
… gene augmentation therapy for Leber congenital amaurosis type 5 (LCA5). … FDA. About Leber Congenital Amaurosis (LCA) and LCA5 Leber congenital amaurosis (LCA) is … inherited retinal diseases including Leber congenital amaurosis (LCA), bestrophinopathy, and …
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Gene therapy for Leber congenital amaurosis has positive phase 1/2 results at 12 months
26 Jun 2025 21:06 GMT
… -LCA5, a gene therapy for Leber congenital amaurosis.
She presented 12-month results … -LCA5 for the treatment of Leber congenital amaurosis, as Healio previously reported.
Sources …
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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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