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Methodological Challenges in the Economic Evaluation of a Gene Therapy for RPE65-Mediated Inherited Retinal Disease: The Value of Vision

The emergence of gene therapies challenge health economists to evaluate interventions that are often provided to a small patient population with a specific gene mutation in a single dose with high upfront costs and uncertain long-term benefits. The objective of this study was to illustrate the metho...

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Detalles Bibliográficos
Autores principales:	Huygens, Simone A., Versteegh, Matthijs M., Vegter, Stefan, Schouten, L. Jan, Kanters, Tim A.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Springer International Publishing 2021
Materias:	Review Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8009797/ https://www.ncbi.nlm.nih.gov/pubmed/33604870 http://dx.doi.org/10.1007/s40273-021-01003-y

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