Short-Term Outcomes of Pediatric Patients With Mild Autosomal Recessive RPE65-Associated Retinal Dystrophy Treated With Voretigene Neparvovec.

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Title: Short-Term Outcomes of Pediatric Patients With Mild Autosomal Recessive RPE65-Associated Retinal Dystrophy Treated With Voretigene Neparvovec.
Authors: Merle DA; Department of Ophthalmology, University Eye Clinic, Eberhard Karls University of Tübingen, Tübingen, Germany.; Institute for Ophthalmic Research, Department for Ophthalmology, Eberhard Karls University of Tübingen, Tübingen, Germany.; Department of Ophthalmology, Medical University of Graz, Graz, Austria., Hertens L; Department of Ophthalmology, Ghent University Hospital, Ghent, Belgium., Dimopoulos S; Department of Ophthalmology, University Eye Clinic, Eberhard Karls University of Tübingen, Tübingen, Germany., Kohl S; Institute for Ophthalmic Research, Department for Ophthalmology, Eberhard Karls University of Tübingen, Tübingen, Germany., Van Haute M; Department of Ophthalmology, Ghent University Hospital, Ghent, Belgium., De Baere E; Center for Medical Genetics Ghent, Ghent University Hospital, Ghent, Belgium.; Department of Biomolecular Medicine, Ghent University, Ghent, Belgium., De Bruyne M; Center for Medical Genetics Ghent, Ghent University Hospital, Ghent, Belgium., Janssens B; Ophthalmological Practice Hippocrates, Sint-Niklaas, Belgium., Rüther K; Ophthalmological Practice Berlin-Mitte, Berlin, Germany., Huchzermeyer C; Department of Ophthalmology, University Hospital Erlangen, Friedrich-Alexander-University Erlangen-Nürnberg, Erlangen, Germany., Mazzola P; Institute for Medical Genetics and Applied Genomics, University Hospital, Eberhard Karls University Tübingen, Tübingen, Germany., Nerinckx F; Department of Ophthalmology, Ghent University Hospital, Ghent, Belgium., Haack T; Institute for Medical Genetics and Applied Genomics, University Hospital, Eberhard Karls University Tübingen, Tübingen, Germany., Wolfram L; Department of Ophthalmology, University Eye Clinic, Eberhard Karls University of Tübingen, Tübingen, Germany.; Institute for Ophthalmic Research, Department for Ophthalmology, Eberhard Karls University of Tübingen, Tübingen, Germany., Kempf M; Department of Ophthalmology, University Eye Clinic, Eberhard Karls University of Tübingen, Tübingen, Germany.; Center for Rare Eye Diseases, Eberhard Karls University of Tübingen, Tübingen, Germany., Kühlewein L; Department of Ophthalmology, University Eye Clinic, Eberhard Karls University of Tübingen, Tübingen, Germany.; Institute for Ophthalmic Research, Department for Ophthalmology, Eberhard Karls University of Tübingen, Tübingen, Germany., Stingl K; Department of Ophthalmology, University Eye Clinic, Eberhard Karls University of Tübingen, Tübingen, Germany.; Center for Rare Eye Diseases, Eberhard Karls University of Tübingen, Tübingen, Germany., Leroy BP; Department of Ophthalmology, Ghent University Hospital, Ghent, Belgium.; Center for Medical Genetics Ghent, Ghent University Hospital, Ghent, Belgium.; Department of Head and Skin, Ghent University, Ghent, Belgium., Stingl K; Department of Ophthalmology, University Eye Clinic, Eberhard Karls University of Tübingen, Tübingen, Germany.; Center for Rare Eye Diseases, Eberhard Karls University of Tübingen, Tübingen, Germany.
Source: Translational vision science & technology [Transl Vis Sci Technol] 2025 Aug 01; Vol. 14 (8), pp. 8.
Publication Type: Journal Article; Case Reports
Journal Info: Publisher: Association for Research in Vision and Ophthalmology Country of Publication: United States NLM ID: 101595919 Publication Model: Print Cited Medium: Internet ISSN: 2164-2591 (Electronic) Linking ISSN: 21642591 NLM ISO Abbreviation: Transl Vis Sci Technol Subsets: MEDLINE
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  Data: Short-Term Outcomes of Pediatric Patients With Mild Autosomal Recessive RPE65-Associated Retinal Dystrophy Treated With Voretigene Neparvovec.
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  Data: <searchLink fieldCode="AU" term="%22Merle+DA%22">Merle DA</searchLink>; Department of Ophthalmology, University Eye Clinic, Eberhard Karls University of Tübingen, Tübingen, Germany.; Institute for Ophthalmic Research, Department for Ophthalmology, Eberhard Karls University of Tübingen, Tübingen, Germany.; Department of Ophthalmology, Medical University of Graz, Graz, Austria.<br /><searchLink fieldCode="AU" term="%22Hertens+L%22">Hertens L</searchLink>; Department of Ophthalmology, Ghent University Hospital, Ghent, Belgium.<br /><searchLink fieldCode="AU" term="%22Dimopoulos+S%22">Dimopoulos S</searchLink>; Department of Ophthalmology, University Eye Clinic, Eberhard Karls University of Tübingen, Tübingen, Germany.<br /><searchLink fieldCode="AU" term="%22Kohl+S%22">Kohl S</searchLink>; Institute for Ophthalmic Research, Department for Ophthalmology, Eberhard Karls University of Tübingen, Tübingen, Germany.<br /><searchLink fieldCode="AU" term="%22Van+Haute+M%22">Van Haute M</searchLink>; Department of Ophthalmology, Ghent University Hospital, Ghent, Belgium.<br /><searchLink fieldCode="AU" term="%22De+Baere+E%22">De Baere E</searchLink>; Center for Medical Genetics Ghent, Ghent University Hospital, Ghent, Belgium.; Department of Biomolecular Medicine, Ghent University, Ghent, Belgium.<br /><searchLink fieldCode="AU" term="%22De+Bruyne+M%22">De Bruyne M</searchLink>; Center for Medical Genetics Ghent, Ghent University Hospital, Ghent, Belgium.<br /><searchLink fieldCode="AU" term="%22Janssens+B%22">Janssens B</searchLink>; Ophthalmological Practice Hippocrates, Sint-Niklaas, Belgium.<br /><searchLink fieldCode="AU" term="%22Rüther+K%22">Rüther K</searchLink>; Ophthalmological Practice Berlin-Mitte, Berlin, Germany.<br /><searchLink fieldCode="AU" term="%22Huchzermeyer+C%22">Huchzermeyer C</searchLink>; Department of Ophthalmology, University Hospital Erlangen, Friedrich-Alexander-University Erlangen-Nürnberg, Erlangen, Germany.<br /><searchLink fieldCode="AU" term="%22Mazzola+P%22">Mazzola P</searchLink>; Institute for Medical Genetics and Applied Genomics, University Hospital, Eberhard Karls University Tübingen, Tübingen, Germany.<br /><searchLink fieldCode="AU" term="%22Nerinckx+F%22">Nerinckx F</searchLink>; Department of Ophthalmology, Ghent University Hospital, Ghent, Belgium.<br /><searchLink fieldCode="AU" term="%22Haack+T%22">Haack T</searchLink>; Institute for Medical Genetics and Applied Genomics, University Hospital, Eberhard Karls University Tübingen, Tübingen, Germany.<br /><searchLink fieldCode="AU" term="%22Wolfram+L%22">Wolfram L</searchLink>; Department of Ophthalmology, University Eye Clinic, Eberhard Karls University of Tübingen, Tübingen, Germany.; Institute for Ophthalmic Research, Department for Ophthalmology, Eberhard Karls University of Tübingen, Tübingen, Germany.<br /><searchLink fieldCode="AU" term="%22Kempf+M%22">Kempf M</searchLink>; Department of Ophthalmology, University Eye Clinic, Eberhard Karls University of Tübingen, Tübingen, Germany.; Center for Rare Eye Diseases, Eberhard Karls University of Tübingen, Tübingen, Germany.<br /><searchLink fieldCode="AU" term="%22Kühlewein+L%22">Kühlewein L</searchLink>; Department of Ophthalmology, University Eye Clinic, Eberhard Karls University of Tübingen, Tübingen, Germany.; Institute for Ophthalmic Research, Department for Ophthalmology, Eberhard Karls University of Tübingen, Tübingen, Germany.<br /><searchLink fieldCode="AU" term="%22Stingl+K%22">Stingl K</searchLink>; Department of Ophthalmology, University Eye Clinic, Eberhard Karls University of Tübingen, Tübingen, Germany.; Center for Rare Eye Diseases, Eberhard Karls University of Tübingen, Tübingen, Germany.<br /><searchLink fieldCode="AU" term="%22Leroy+BP%22">Leroy BP</searchLink>; Department of Ophthalmology, Ghent University Hospital, Ghent, Belgium.; Center for Medical Genetics Ghent, Ghent University Hospital, Ghent, Belgium.; Department of Head and Skin, Ghent University, Ghent, Belgium.<br /><searchLink fieldCode="AU" term="%22Stingl+K%22">Stingl K</searchLink>; Department of Ophthalmology, University Eye Clinic, Eberhard Karls University of Tübingen, Tübingen, Germany.; Center for Rare Eye Diseases, Eberhard Karls University of Tübingen, Tübingen, Germany.
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