12-month outcomes after voretigene neparvovec gene therapy in paediatric patients with<i>RPE65</i>-mediated inherited retinal dystrophy
Most paediatric patients treated by voretigene neparvovec showed a significant increase in visual function at 12 months follow-up. None of the postoperative complications prevented gains in visual function.
Authors
- Alejandra Daruich (ORCID: https://orcid.org/0000-0001-7907-4325)
- Maxence Rateaux
- Emilie Batté
- Sophie Valleix (ORCID: https://orcid.org/0000-0002-4812-7102)
- Matthieu P. Robert (ORCID: https://orcid.org/0000-0003-3353-8117)
- Dominique Brémond‐Gignac (ORCID: https://orcid.org/0000-0002-3563-5482)
- Nathalie de Vergnes
Institutions
- Hôpital Necker-Enfants Malades (FR)
- Centre National de la Recherche Scientifique (FR)
- Inserm (FR)
- Université Paris Cité (FR)
- Sorbonne Paris Cité (FR)
- Centre de Recherche des Cordeliers (FR)
- Assistance Publique – Hôpitaux de Paris (FR)
Publication Details
- Journal
- British Journal of Ophthalmology
- Published
- 2024-11-21
- DOI
- https://doi.org/10.1136/bjo-2024-326221
- Citations
- 11
- Primary Topic
- Retinal Development and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 2.57