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RPE65-associated inherited retinal diseases: consensus recommendations for eligibility to gene therapy
BACKGROUND: This research aimed to establish recommendations on the clinical and genetic characteristics necessary to confirm patient eligibility for gene supplementation with voretigene neparvovec. METHODS: An expert steering committee comprising an interdisciplinary panel of Italian experts in the...
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| Publicado no: | Orphanet J Rare Dis |
|---|---|
| Main Authors: | , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
BioMed Central
2021
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC8176684/ https://ncbi.nlm.nih.gov/pubmed/34088339 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-021-01868-4 |
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