Short-Term Outcomes of Pediatric Patients With Mild Autosomal Recessive <i>RPE65</i>-Associated Retinal Dystrophy Treated With Voretigene Neparvovec
This study aids clinical decision-making in unclear cases by demonstrating that early treatment with voretigene neparvovec in mild RPE65-IRD provides functional benefits while minimizing the risk of fast-growing chorioretinal atrophy.
Authors
- Lasse Wolfram (ORCID: https://orcid.org/0000-0003-1549-5889)
- Tobias B. Haack (ORCID: https://orcid.org/0000-0001-6033-4836)
- Susanne Kohl (ORCID: https://orcid.org/0000-0002-6438-6331)
- Bart P. Leroy (ORCID: https://orcid.org/0000-0002-9899-2081)
- Klaus Rüther
- Fanny Nerinckx
- Laura Kuehlewein (ORCID: https://orcid.org/0000-0003-1906-8681)
- Pascale Mazzola (ORCID: https://orcid.org/0000-0002-2971-5603)
- David A. Merle (ORCID: https://orcid.org/0000-0003-4485-5580)
- Cord Huchzermeyer (ORCID: https://orcid.org/0000-0002-0408-8981)
- Katarína Štingl (ORCID: https://orcid.org/0000-0002-8132-911X)
- Melanie Kempf (ORCID: https://orcid.org/0000-0002-0370-0462)
- Elfride De Baere (ORCID: https://orcid.org/0000-0002-5609-6895)
- Marieke De Bruyne (ORCID: https://orcid.org/0000-0001-6636-5537)
- Spyridon Dimopoulos (ORCID: https://orcid.org/0000-0002-5074-7300)
- Barbara Janssens (ORCID: https://orcid.org/0009-0009-5960-6457)
- Krunoslav Stingl
- Manon van Haute
- Leen Hertens
Institutions
- Friedrich-Alexander-Universität Erlangen-Nürnberg (DE)
- Medical University of Graz (AT)
- Berlin Zoological Garden (DE)
- Ghent University Hospital (BE)
- Ghent University (BE)
- Universitätsklinikum Erlangen (DE)
- Vlaamse Vereniging voor Obstetrie en Gynaecolo (BE)
- STZ eyetrial (DE)
- University of Tübingen (DE)
Publication Details
- Journal
- Translational Vision Science & Technology
- Published
- 2025-08-04
- DOI
- https://doi.org/10.1167/tvst.14.8.8
- Citations
- 2
- Primary Topic
- Retinal Development and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 1.61
Funders
- Biogen
- REGENXBIO
- Eberhard Karls Universität Tübingen
- Fonds Wetenschappelijk Onderzoek
- Universitair Ziekenhuis Gent
- Santen
- Universiteit Gent
- Vlaamse regering
- H. Lundbeck A/S
- ProQR Therapeutics
- Spark Therapeutics
- Janssen Pharmaceuticals