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[Implementation of a new gene therapy in ophthalmology: Regulatory and organizational issues]
A Daruich1, R Boinet2, C Falcou3
1Service d'ophtalmologie, hôpital Necker-Enfants Malades, université de Paris, AP-HP, OPHTARA Center, 149, rue de Sèvres, Paris, France; Inserm, UMRS1138, équipe 17, centre de recherche des Cordeliers, 15, rue de l'École de Médecine, Paris, France.
Abstract:
Voretigene neparvovec (VN) is the first gene therapy in ophthalmology for patients with RPE65-mediated hereditary retinal dystrophy. It has recently obtained European market approval, which is subject to strict regulatory and organizational conditions for its use. Here, we analyze the main studies supporting the authorization of this new therapy and describe the necessary steps to take at a hospital level for optimal administration to patients following current regulations.
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