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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Detalles Bibliográficos
Publicado en:Orphanet J Rare Dis
Main Authors: Sodi, Andrea, Banfi, Sandro, Testa, Francesco, Della Corte, Michele, Passerini, Ilaria, Pelo, Elisabetta, Rossi, Settimio, Simonelli, Francesca
Formato: Artigo
Idioma:Inglês
Publicado: BioMed Central 2021
Assuntos:
Research
Acceso en liña: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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