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J. Hipp, C. Bacino, L. Bird, Ina Bruenig-Traebert, D.E.C.Y. Chan, Marie-Claire Y. de Wit, Paulo Fontoura, G. Hooper, Ravi Jagasia, Michelle L. Krishnan, Lorraine Murtagh, Alessandro Noci, Ana Roche Martínez, Dietmar Schwab, Mercedes Serrano, Mark D. Shen, J. Tillmann, J. Tjeertes, B. Vincenzi, E. Berry-Kravis, Azad Bonni, M. Barakovic, Charlotte Bon, Ponghatai Boonsimma, M. Bustamante, Katalin Buzasi, Stormy Chamberlain, Susanne Clinch, Siobhan Connor-Ahmad, Philipp Daetwyler, Oana Danila, S. Holst, Thomas Kremer, F. Lipsmeier, S. Magon, A. Messer, David Nobbs, Giuseppe Palermo, Lisa Squassante, Thomas Wiese, Jennifer M. Bain, Jamie K. Capal, R. Gavrilova, M. M. Cabeza, N. Pietrafusa, Rajsekar Rajamaran, S. Jeste, F. Vigevano
24 11. 7. 2025.

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


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