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Advanced filters: Author: Malgorzata Swider Clear advanced filters

Durable vision improvement after a single treatment with antisense oligonucleotide sepofarsen: a case report

Post hoc analyses and follow-up of a patient with Leber congenital amaurosis treated with the antisense oligonucleotide sepofarsen as part of a clinical trial indicates sustained improvement of vision 15 months after receiving a single dose of the drug.

Artur V. Cideciyan
Samuel G. Jacobson
Aniz Girach
Research01 Apr 2021
Nature Medicine

Volume: 27, P: 785-789
Retinal prolactin isoform PRLΔE1 sustains rod disease in inherited retinal degenerations

Raghavi Sudharsan
Jennifer Kwok
William A. Beltran
ResearchOpen Access18 Sept 2024
Cell Death & Disease

Volume: 15, P: 1-10
Rod function deficit in retained photoreceptors of patients with class B Rhodopsin mutations

Artur V. Cideciyan
Samuel G. Jacobson
William A. Beltran
ResearchOpen Access28 Jul 2020
Scientific Reports

Volume: 10, P: 1-15