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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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| Publicado no: | Eye (Lond) |
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| Principais autores: | , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Nature Publishing Group UK
2019
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| Assuntos: | |
| Acesso em linha: | 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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