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
Description
ISSN:1546-170X
DOI:10.1038/s41591-025-03784-7