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Estimation of impact of RPE65-mediated inherited retinal disease on quality of life and the potential benefits of gene therapy

BACKGROUND/AIMS: In rare diseases, health-related quality of life (HRQL) data can be difficult to capture. Given the ultrarare nature of RPE65-mediated inherited retinal disease (IRD), it was not feasible to recruit a patient sample and collect HRQL data prospectively. The objectives of this study w...

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Detalles Bibliográficos
Autores principales:	Lloyd, Andrew, Piglowska, Natalia, Ciulla, Thomas, Pitluck, Sarah, Johnson, Scott, Buessing, Marric, O’Connell, Thomas
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BMJ Publishing Group 2019
Materias:	Clinical Science
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6855782/ https://www.ncbi.nlm.nih.gov/pubmed/30658988 http://dx.doi.org/10.1136/bjophthalmol-2018-313089

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