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Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial.
Nat Med. 2022 May;28(5):1014-1021. doi: 10.1038/s41591-022-01755-w. Epub 2022 Apr 4.
Nat Med. 2022.
PMID: 35379979
Free PMC article.
Clinical Trial.
LEBER CONGENITAL AMAUROSIS DUE TO CEP290 MUTATIONS-SEVERE VISION IMPAIRMENT WITH A HIGH UNMET MEDICAL NEED: A Review.
Leroy BP, Birch DG, Duncan JL, Lam BL, Koenekoop RK, Porto FBO, Russell SR, Girach A.
Leroy BP, et al.
Retina. 2021 May 1;41(5):898-907. doi: 10.1097/IAE.0000000000003133.
Retina. 2021.
PMID: 33595255
Free PMC article.
Review.
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