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Gene and Cell Therapies Hold Potential—but How Can Payers Manage Their Costs?
18 Apr 2024 22:39 GMT
… regarding an individual diagnosed with Leber congenital amaurosis, a retinal dystrophy the severely …
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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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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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Atsena Therapeutics Announces Positive 12-month Safety and Efficacy Data from Ongoing Phase I/II Clinical Trial of ATSN-101 in Patients with Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D (LCA1)
04 Dec 2023 12:30 GMT
… the treatment of GUCY2D-associated Leber congenital amaurosis (LCA1). At 12 months … of LCA1. About GUCY2D-associated Leber congenital amaurosis (LCA1)
LCA1 is a monogenic … ATSN-101 for GUCY2D-associated Leber congenital amaurosis (LCA1). ATSN-201, which …
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Traditional gene therapies are uber-niche. Ocugen hopes to change that.
15 Apr 2024 14:20 GMT
… inherited retinal diseases: RP and Leber congenital amaurosis. It works by delivering a …
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Kiora Pharmaceuticals Receives Funding for Inherited Retinal Disease Trials
15 Apr 2024 11:18 GMT
… macula, which damages retinal cells.
● Leber Congenital Amaurosis (LCA): A severe genetic disorder …
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Atsena Therapeutics Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for ATSN-101 Gene Therapy for GUCY2D-associated Leber Congenital Amaurosis (LCA1)
14 Nov 2023 12:30 GMT
… gene therapy for patients with Leber congenital amaurosis caused by biallelic mutations in … of LCA1. About GUCY2D-associated Leber congenital amaurosis (LCA1)
LCA1 is a … ATSN-101 for GUCY2D-associated Leber congenital amaurosis (LCA1). ATSN-201, which …
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Living in the Dark: A Look into Leber Congenital Amaurosis
19 Oct 2023 11:50 GMT
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Signs and Symptoms of Leber Congenital Amaurosis
Symptoms of LCA usually … British Journal of Ophthalmology
Can Leber Congenital Amaurosis Be Cured?
Currently, there … treatment.
Conclusion
In conclusion, Leber Congenital Amaurosis is a rare genetic disorder …
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Toddler suffering from ‘one-in-a-million’ Leber congenital amaurosis takes medical leap of faith
07 Oct 2023 16:40 GMT
… treatment.
Ciro Carrascull suffers from Leber congenital amaurosis (LCA), a rare inherited retinal …