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The UBE3A-ATS antisense oligonucleotide rugonersen in children with Angelman syndrome: a phase 1 trial.

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Title: The UBE3A-ATS antisense oligonucleotide rugonersen in children with Angelman syndrome: a phase 1 trial.
Authors: Hipp JF; Roche Pharma Research and Early Development, Neuroscience and Rare Diseases, Roche Innovation Center Basel, F. Hoffmann-La Roche, Basel, Switzerland. joerg.hipp@roche.com., Bacino CA; Department of Molecular and Human Genetics, Baylor College of Medicine and Baylor Genetics Laboratory, Texas Children's Hospital, Houston, TX, USA., Bird LM; Department of Pediatrics; Division of Genetics/Dysmorphology, Rady Children's Hospital San Diego, University of California San Diego, San Diego, USA., Bruenig-Traebert I; Roche Pharma Research and Early Development, Neuroscience and Rare Diseases, Roche Innovation Center Basel, F. Hoffmann-La Roche, Basel, Switzerland., Chan D; Roche Pharma Research and Early Development (pRED), Strategy, Portfolio & Operations, Roche Innovation Center Basel, Basel, Switzerland., de Wit MC; Department of Neurology and Pediatric Neurology and Erasmus MC center of expertise for neurodevelopmental disorders ENCORE, Erasmus MC, Rotterdam, The Netherlands., Fontoura P; NOVA Medical School, Lisbon, Portugal., Hooper G; Roche Products Ltd, Welwyn Garden City, UK., Jagasia R; Roche Pharma Research and Early Development, Neuroscience and Rare Diseases, Roche Innovation Center Basel, F. Hoffmann-La Roche, Basel, Switzerland., Krishnan ML; Roche Pharma Research and Early Development, Neuroscience and Rare Diseases, Roche Innovation Center Basel, F. Hoffmann-La Roche, Basel, Switzerland., Murtagh L; Roche Pharma Research and Early Development, Neuroscience and Rare Diseases, Roche Innovation Center Basel, F. Hoffmann-La Roche, Basel, Switzerland., Noci A; Product Development Neuroscience, Roche Innovation Center Basel, F. Hoffmann-La Roche, Basel, Switzerland., Martínez AR; Division of Pediatric, Parc Taulí Hospital Universitari. Institut d'Investigació i Innovació Parc Taulí (I3PT-CERCA), Sabadell, Spain., Schwab D; Roche Pharma Research and Early Development, Pharmaceutical Sciences, Roche Innovation Center Basel, F. Hoffmann-La Roche, Basel, Switzerland., Serrano M; Pediatric Neurology Department, Hospital Sant Joan de Déu, Institut de Recerca Sant Joan de Déu, Barcelona, Spain., Shen MD; UNC Carolina Institute for Developmental Disabilities (CIDD), Dept. of Psychiatry, and Neuroscience Center, University of North Carolina School of Medicine, Chapel Hill, NC, USA., Tillmann J; Roche Pharma Research and Early Development, Neuroscience and Rare Diseases, Roche Innovation Center Basel, F. Hoffmann-La Roche, Basel, Switzerland., Tjeertes J; Product Development Neuroscience, Roche Innovation Center Basel, F. Hoffmann-La Roche, Basel, Switzerland., Vincenzi B; Product Development Neuroscience, Roche Innovation Center Basel, F. Hoffmann-La Roche, Basel, Switzerland., Berry-Kravis E; Departments of Pediatrics, Neurological Sciences, Anatomy and Cell Biology, Rush University Medical Center, Chicago, IL, USA., Bonni A; Roche Pharma Research and Early Development, Neuroscience and Rare Diseases, Roche Innovation Center Basel, F. Hoffmann-La Roche, Basel, Switzerland.
Corporate Authors: Rugonersen Study Group, all TANGELO investigators
Source: Nature medicine [Nat Med] 2025 Sep; Vol. 31 (9), pp. 2936-2945. Date of Electronic Publication: 2025 Jul 11.
Publication Type: Journal Article; Clinical Trial, Phase I; Multicenter Study
Journal Info: Publisher: Nature Publishing Company Country of Publication: United States NLM ID: 9502015 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1546-170X (Electronic) Linking ISSN: 10788956 NLM ISO Abbreviation: Nat Med Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: The UBE3A-ATS antisense oligonucleotide rugonersen in children with Angelman syndrome: a phase 1 trial.
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  Data: <searchLink fieldCode="AU" term="%22Hipp+JF%22">Hipp JF</searchLink>; Roche Pharma Research and Early Development, Neuroscience and Rare Diseases, Roche Innovation Center Basel, F. Hoffmann-La Roche, Basel, Switzerland. joerg.hipp@roche.com.<br /><searchLink fieldCode="AU" term="%22Bacino+CA%22">Bacino CA</searchLink>; Department of Molecular and Human Genetics, Baylor College of Medicine and Baylor Genetics Laboratory, Texas Children's Hospital, Houston, TX, USA.<br /><searchLink fieldCode="AU" term="%22Bird+LM%22">Bird LM</searchLink>; Department of Pediatrics; Division of Genetics/Dysmorphology, Rady Children's Hospital San Diego, University of California San Diego, San Diego, USA.<br /><searchLink fieldCode="AU" term="%22Bruenig-Traebert+I%22">Bruenig-Traebert I</searchLink>; Roche Pharma Research and Early Development, Neuroscience and Rare Diseases, Roche Innovation Center Basel, F. Hoffmann-La Roche, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Chan+D%22">Chan D</searchLink>; Roche Pharma Research and Early Development (pRED), Strategy, Portfolio & Operations, Roche Innovation Center Basel, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22de+Wit+MC%22">de Wit MC</searchLink>; Department of Neurology and Pediatric Neurology and Erasmus MC center of expertise for neurodevelopmental disorders ENCORE, Erasmus MC, Rotterdam, The Netherlands.<br /><searchLink fieldCode="AU" term="%22Fontoura+P%22">Fontoura P</searchLink>; NOVA Medical School, Lisbon, Portugal.<br /><searchLink fieldCode="AU" term="%22Hooper+G%22">Hooper G</searchLink>; Roche Products Ltd, Welwyn Garden City, UK.<br /><searchLink fieldCode="AU" term="%22Jagasia+R%22">Jagasia R</searchLink>; Roche Pharma Research and Early Development, Neuroscience and Rare Diseases, Roche Innovation Center Basel, F. Hoffmann-La Roche, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Krishnan+ML%22">Krishnan ML</searchLink>; Roche Pharma Research and Early Development, Neuroscience and Rare Diseases, Roche Innovation Center Basel, F. Hoffmann-La Roche, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Murtagh+L%22">Murtagh L</searchLink>; Roche Pharma Research and Early Development, Neuroscience and Rare Diseases, Roche Innovation Center Basel, F. Hoffmann-La Roche, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Noci+A%22">Noci A</searchLink>; Product Development Neuroscience, Roche Innovation Center Basel, F. Hoffmann-La Roche, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Martínez+AR%22">Martínez AR</searchLink>; Division of Pediatric, Parc Taulí Hospital Universitari. Institut d'Investigació i Innovació Parc Taulí (I3PT-CERCA), Sabadell, Spain.<br /><searchLink fieldCode="AU" term="%22Schwab+D%22">Schwab D</searchLink>; Roche Pharma Research and Early Development, Pharmaceutical Sciences, Roche Innovation Center Basel, F. Hoffmann-La Roche, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Serrano+M%22">Serrano M</searchLink>; Pediatric Neurology Department, Hospital Sant Joan de Déu, Institut de Recerca Sant Joan de Déu, Barcelona, Spain.<br /><searchLink fieldCode="AU" term="%22Shen+MD%22">Shen MD</searchLink>; UNC Carolina Institute for Developmental Disabilities (CIDD), Dept. of Psychiatry, and Neuroscience Center, University of North Carolina School of Medicine, Chapel Hill, NC, USA.<br /><searchLink fieldCode="AU" term="%22Tillmann+J%22">Tillmann J</searchLink>; Roche Pharma Research and Early Development, Neuroscience and Rare Diseases, Roche Innovation Center Basel, F. Hoffmann-La Roche, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Tjeertes+J%22">Tjeertes J</searchLink>; Product Development Neuroscience, Roche Innovation Center Basel, F. Hoffmann-La Roche, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Vincenzi+B%22">Vincenzi B</searchLink>; Product Development Neuroscience, Roche Innovation Center Basel, F. Hoffmann-La Roche, Basel, Switzerland.<br /><searchLink fieldCode="AU" term="%22Berry-Kravis+E%22">Berry-Kravis E</searchLink>; Departments of Pediatrics, Neurological Sciences, Anatomy and Cell Biology, Rush University Medical Center, Chicago, IL, USA.<br /><searchLink fieldCode="AU" term="%22Bonni+A%22">Bonni A</searchLink>; Roche Pharma Research and Early Development, Neuroscience and Rare Diseases, Roche Innovation Center Basel, F. Hoffmann-La Roche, Basel, Switzerland.
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  Data: <searchLink fieldCode="JN" term="%229502015%22">Nature medicine</searchLink> [Nat Med] 2025 Sep; Vol. 31 (9), pp. 2936-2945. <i>Date of Electronic Publication: </i>2025 Jul 11.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Nature+Publishing+Company%22">Nature Publishing Company </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>9502015 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1546-170X (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2210788956%22">10788956 </searchLink><i>NLM ISO Abbreviation: </i>Nat Med <i>Subsets: </i>MEDLINE
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