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Potential lifetime quality of life benefits of choroideremia gene therapy: projections from a clinically informed decision model

BACKGROUND: The first gene therapy for an inherited retinal dystrophy recently received market approval in the United States; multiple other gene therapies are in the clinical pipeline. Thus far, gene therapy has commanded prices in the range of $500,000 to over $1,000,000 for the one-time doses and...

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Dettagli Bibliografici
Pubblicato in:Eye (Lond)
Autori principali: Halioua-Haubold, Celine-Lea, Jolly, Jasleen K., Smith, James A., Pinedo-Villanueva, Rafael, Brindley, David A., MacLaren, Robert E.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group UK 2019
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7005854/
https://ncbi.nlm.nih.gov/pubmed/31312000
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41433-019-0492-1
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