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. |
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| 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 |
| Database: | MEDLINE Ultimate |
| FullText | Text: Availability: 0 |
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| Header | DbId: mdl DbLabel: MEDLINE Ultimate An: 40757766 AccessLevel: 2 PubType: Academic Journal PubTypeId: academicJournal PreciseRelevancyScore: 0 |
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| Items | – Name: Title Label: Title Group: Ti Data: Short-Term Outcomes of Pediatric Patients With Mild Autosomal Recessive RPE65-Associated Retinal Dystrophy Treated With Voretigene Neparvovec. – Name: Author Label: Authors Group: Au 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. – Name: TitleSource Label: Source Group: Src Data: <searchLink fieldCode="JN" term="%22101595919%22">Translational vision science & technology</searchLink> [Transl Vis Sci Technol] 2025 Aug 01; Vol. 14 (8), pp. 8. – Name: TypePub Label: Publication Type Group: TypPub Data: Journal Article; Case Reports – Name: TitleSource Label: Journal Info Group: Src Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Association+for+Research+in+Vision+and+Ophthalmology%22">Association for Research in Vision and Ophthalmology </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>101595919 <i>Publication Model: </i>Print <i>Cited Medium: </i>Internet <i>ISSN: </i>2164-2591 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2221642591%22">21642591 </searchLink><i>NLM ISO Abbreviation: </i>Transl Vis Sci Technol <i>Subsets: </i>MEDLINE |
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| RecordInfo | BibRecord: BibEntity: Identifiers: – Type: doi Value: 10.1167/tvst.14.8.8 Languages: – Code: eng Text: English PhysicalDescription: Pagination: StartPage: 8 Titles: – TitleFull: Short-Term Outcomes of Pediatric Patients With Mild Autosomal Recessive RPE65-Associated Retinal Dystrophy Treated With Voretigene Neparvovec. Type: main BibRelationships: HasContributorRelationships: – PersonEntity: Name: NameFull: Merle DA – PersonEntity: Name: NameFull: Hertens L – PersonEntity: Name: NameFull: Dimopoulos S – PersonEntity: Name: NameFull: Kohl S – PersonEntity: Name: NameFull: Van Haute M – PersonEntity: Name: NameFull: De Baere E – PersonEntity: Name: NameFull: De Bruyne M – PersonEntity: Name: NameFull: Janssens B – PersonEntity: Name: NameFull: Rüther K – PersonEntity: Name: NameFull: Huchzermeyer C – PersonEntity: Name: NameFull: Mazzola P – PersonEntity: Name: NameFull: Nerinckx F – PersonEntity: Name: NameFull: Haack T – PersonEntity: Name: NameFull: Wolfram L – PersonEntity: Name: NameFull: Kempf M – PersonEntity: Name: NameFull: Kühlewein L – PersonEntity: Name: NameFull: Stingl K – PersonEntity: Name: NameFull: Leroy BP – PersonEntity: Name: NameFull: Stingl K IsPartOfRelationships: – BibEntity: Dates: – D: 01 M: 08 Text: 2025 Aug 01 Type: published Y: 2025 Identifiers: – Type: issn-electronic Value: 2164-2591 Numbering: – Type: volume Value: 14 – Type: issue Value: 8 Titles: – TitleFull: Translational vision science & technology Type: main |
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