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Late presentation of RPE65 retinopathy in three siblings

PURPOSE: Gene therapy for RPE65 retinopathy has been recently approved. The purpose of this study was to assess retinal structure and function in 3 siblings presenting with late-stage RPE65 retinopathy and to assess the unmet need for such therapy in Saudi Arabia. METHODS: Search of the retinal dyst...

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Detalles Bibliográficos
Autores principales: Magliyah, Moustafa, Saifaldein, Amjad Ameen, Schatz, Patrik
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Berlin Heidelberg 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7205780/
https://www.ncbi.nlm.nih.gov/pubmed/31925606
http://dx.doi.org/10.1007/s10633-019-09745-z