Effects of tofersen treatment in patients with SOD1-ALS in a “real-world” setting – a 12-month multicenter cohort study from the German early access programResearch in context

Effects of tofersen treatment in patients with SOD1-ALS in a “real-world” setting – a 12-month multicenter cohort study from the German early access programResearch in context

Summary: Background: In April 2023, the antisense oligonucleotide tofersen was approved by the U.S. Food and Drug Administration (FDA) for treatment of SOD1-amyotrophic lateral sclerosis (ALS), after a decrease of neurofilament light chain (NfL) levels had been demonstrated. Methods: Between 03/202...

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Hauptverfasser: Maximilian Wiesenfarth, Johannes Dorst, David Brenner, Zeynep Elmas, Özlem Parlak, Zeljko Uzelac, Katharina Kandler, Kristina Mayer, Ulrike Weiland, Christine Herrmann, Joachim Schuster, Axel Freischmidt, Kathrin Müller, Reiner Siebert, Franziska Bachhuber, Tatiana Simak, Kornelia Günther, Elke Fröhlich, Antje Knehr, Martin Regensburger, Alexander German, Susanne Petri, Julian Grosskreutz, Thomas Klopstock, Peter Reilich, Florian Schöberl, Tim Hagenacker, Ute Weyen, René Günther, Maximilian Vidovic, Martin Jentsch, Thomas Haarmeier, Patrick Weydt, Ivan Valkadinov, Jasper Hesebeck-Brinckmann, Julian Conrad, Jochen Hans Weishaupt, Peggy Schumann, Peter Körtvélyessy, Thomas Meyer, Wolfgang Philipp Ruf, Simon Witzel, Makbule Senel, Hayrettin Tumani, Albert Christian Ludolph
Format: Artikel
Sprache:English
Veröffentlicht: Elsevier 2024-03-01
Schriftenreihe:EClinicalMedicine
Schlagworte:
Amyotrophic lateral sclerosis
SOD1
Antisense oligonucleotide
Tofersen
Early access program
Online Zugang:http://www.sciencedirect.com/science/article/pii/S2589537024000749

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http://www.sciencedirect.com/science/article/pii/S2589537024000749

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