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...

Descripción completa

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

Internet

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

Ejemplares similares