Patient reported outcomes in adolescents with inherited retinal diseases
4 October 2023
| Ian Reekie
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Retina / Uvea / Vitreous
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PROM, Patient-reported outcomes, clinical trial, gene therapy, inherited retinal disease, pediatrics, retinal dystrophy
This was a two-centre validation study of two sets of patient reported outcome measures (PROMS) for adolescents (13-17- years-old) with inherited retinal diseases. The measures were the Michigan Retinal Degeneration Questionnaire (MRDQ) and the Michigan Vision-Related Anxiety Questionnaire (MVAQ). Both...
Prognostic factors for visual acuity in patients with LHON after gene therapy
5 February 2020
| Ivan Yip
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Retina / Uvea / Vitreous
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Leber's hereditary optic neuropathy, ND4, gene therapy, prognosis
This retrospective study investigates prognostic factors affecting the visual acuity after a single intravitreal injection of rAAV2-ND4 gene therapy in 53 patients with Leber’s hereditary optic neuropathy (LHON) with mutation at site 11 778. Patients were reviewed one and three...
