| Autor: |
Callie Deng, Peter Y. Zhao, Kari Branham, Dana Schlegel, Abigail T. Fahim, Thiran K. Jayasundera, Naheed Khan, Cagri G. Besirli |
| Jazyk: |
angličtina |
| Rok vydání: |
2022 |
| Předmět: |
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| Zdroj: |
Graefes Arch Clin Exp Ophthalmol |
| Popis: |
PURPOSE: To investigate real-world safety and efficacy of voretigene neparvovec gene therapy administration in pediatric patients with biallelic RPE65 disease-causing variants. METHODS: A retrospective study of 27 eyes of 14 patients with RPE65-associated Leber congenital amaurosis examined post-operative complications and longitudinal changes in photoreceptor function following treatment with subretinal injection of voretigene neparvovec. Full-field stimulus threshold testing (FST), Goldmann visual fields (GVF), best corrected visual acuity (BCVA), and central subfield thickness (CST) on optical coherence tomography (OCT) scans were collected pre-operatively and up to 12 months post-treatment. RESULTS: Baseline through 6-12 month follow-up FST and GVF data were obtained for 13 eyes of 7 patients. FST improved for each eye after treatment with mean improvement of 2.1 log-units (P < .001) and GVF improved for each eye with mean improvement of 221 sum-degrees (P |
| Databáze: |
OpenAIRE |
| Externí odkaz: |
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