Cost-effectiveness of Voretigene Neparvovec-rzyl vs Standard Care for RPE65-Mediated Inherited Retinal Disease

IMPORTANCE: Voretigene neparvovec-rzyl, the first gene therapy approved by the US Food and Drug Administration, was approved for the treatment for RPE65-mediated inherited retinal disease (IRD) in December 2017. This gene therapy is associated with high up-front costs and high efficacy, although of...

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Bibliografiske detaljer
Udgivet i:JAMA Ophthalmol
Main Authors: Johnson, Scott, Buessing, Marric, O’Connell, Thomas, Pitluck, Sarah, Ciulla, Thomas A.
Format: Artigo
Sprog:Inglês
Udgivet: American Medical Association 2019
Fag:
Original Investigation
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6646972/
https://ncbi.nlm.nih.gov/pubmed/31318398
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1001/jamaophthalmol.2019.2512
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