The UBE3A-ATS antisense oligonucleotide rugonersen in children with Angelman syndrome: a phase 1 trial
Year: 2025
Type: article
Study design: Clinical Trial
Source: Nature Medicine
Authors: Jörg F. Hipp, Carlos A. Bacino, Lynne M. Bird, Ina Bruenig-Traebert, Daniel Chan, Marie Claire de Wit, Paulo Fontoura, Gregory Hooper, Ravi Jagasia, Michelle L. Krishnan, Lorraine Murtagh, Alessandro Noci, Ana Roche Martínez, Dietmar Schwab, Mercedes Serrano, Mark D. Shen, Julian Tillmann, Jorrit Tjeertes, Brenda Vincenzi, Elizabeth Berry-Kravis, Azad Bonni, the Rugonersen Study Group, Muhamed Barakovic, Charlotte Bon, Ponghatai Boonsimma, Mariana Bustamante, Katalin Buzasi, Stormy Chamberlain, Susanne Clinch, Siobhan Connor-Ahmad, Philipp Daetwyler, Oana Danila, Sebastian Holst, Thomas Kremer, Florian Lipsmeier, Stefano Magon, Amanda Messer, David Nobbs, Giuseppe Palermo, Lisa Squassante, Thomas Wiese, all TANGELO investigators, Jennifer M. Bain, Jamie Capal, Ralitza Gavrilova, María Muñoz Cabeza, Nicola Pietrafusa, Rajsekar Rajamaran, Shafali Jeste, Federico Vigevano
Institutions: Roche (Switzerland), Baylor College of Medicine, Texas Children's Hospital, University of California San Diego, Rady Children's Hospital-San Diego, Erasmus MC, Universidade Nova de Lisboa, Roche (United Kingdom), Corporació Sanitària Parc Taulí, Institute of Research and Innovation Parc Tauli, Hospital Sant Joan de Déu Barcelona, Institut de Recerca Sant Joan de Déu, University of North Carolina at Chapel Hill, Rush University Medical Center, Morgan Stanley Children's Hospital, Columbia University Irving Medical Center, Mayo Clinic, Hospital Universitario Virgen del Rocío, Bambino Gesù Children's Hospital, University of California, Los Angeles, Mattel Children's Hospital, University of Southern California, Children's Hospital of Los Angeles, Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: green
Funder: F. Hoffmann-La Roche
OpenAlex ID: W4412192244