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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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Successful treatment of RPE65-mediated inherited retinal dystrophy with voretigene neparvovec
31 Jan 2025 16:15 GMT
… , KaushalS, et al. Treatment of Leber congenital amaurosis due to RPE65 mutations by … therapy in patients with RPE65 Leber congenital amaurosis.Mol Ther. 2018;26:256 … neparvovec-rzyl for RPE65-mediated leber congenital amaurosis.Ophthalmol Retina. 2022;6:58 …
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Retinal diseases: RNA therapeutics show promise but is India ready?
30 Jan 2025 00:51 GMT
… retinal conditions like Stargardt disease, Leber congenital amaurosis, and retinitis pigmentosa.
Beyond ASOs …
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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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The struggle in the dark: Navigating night blindness
26 Jan 2025 09:49 GMT
… vessels), congenital disorders such as Leber congenital amaurosis and severe myopia (near-sightedness …
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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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RNIB’s pre-employment team wins ERSA Employability Award for tailored support
24 Jan 2025 11:18 GMT
… Ahmed, 30, was born with Leber congenital amaurosis. He struggled to secure the …
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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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Positive early safety, efficacy data reported for Leber congenital amaurosis gene therapy
06 Dec 2023 15:18 GMT
… gene therapy in GUCY2D-associated Leber congenital amaurosis.
Fifteen patients received unilateral subretinal … gene therapy in GUCY2D-associated Leber congenital amaurosis.
Image: Adobe Stock
There were …
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