Long-term efficacy and safety of nusinersen in adults with 5q spinal muscular atrophy: a prospective European multinational observational studyResearch in context

• Main Authors: René Günther (Author), Claudia Diana Wurster (Author), Svenja Brakemeier (Author), Alma Osmanovic (Author), Olivia Schreiber-Katz (Author), Susanne Petri (Author), Zeljko Uzelac (Author), Miriam Hiebeler (Author), Simone Thiele (Author), Maggie C. Walter (Author), Markus Weiler (Author), Tobias Kessler (Author), Maren Freigang (Author), Hanna Sophie Lapp (Author), Isabell Cordts (Author), Paul Lingor (Author), Marcus Deschauer (Author), Andreas Hahn (Author), Kyriakos Martakis (Author), Robert Steinbach (Author), Benjamin Ilse (Author), Annekathrin Rödiger (Author), Julia Bellut (Author), Julia Nentwich (Author), Daniel Zeller (Author), Mohamad Tareq Muhandes (Author), Tobias Baum (Author), Jan Christoph Koch (Author), Bertold Schrank (Author), Sophie Fischer (Author), Andreas Hermann (Author), Christoph Kamm (Author), Steffen Naegel (Author), Alexander Mensch (Author), Markus Weber (Author), Christoph Neuwirth (Author), Helmar C. Lehmann (Author), Gilbert Wunderlich (Author), Christian Stadler (Author), Maike Tomforde (Author), Annette George (Author), Martin Groß (Author), Astrid Pechmann (Author), Janbernd Kirschner (Author), Matthias Türk (Author), Mareike Schimmel (Author), Günther Bernert (Author), Pascal Martin (Author), Christian Rauscher (Author), Gerd Meyer zu Hörste (Author), Petra Baum (Author), Wolfgang Löscher (Author), Marina Flotats-Bastardas (Author), Cornelia Köhler (Author), Kristina Probst-Schendzielorz (Author), Susanne Goldbach (Author), Ulrike Schara-Schmidt (Author), Wolfgang Müller-Felber (Author), Hanns Lochmüller (Author), Otgonzul von Velsen (Author), Christoph Kleinschnitz (Author), Albert C. Ludolph (Author), Tim Hagenacker (Author)
• Format: Book
• Published: Elsevier, 2024-04-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Summary: Background: Evidence for the efficacy of nusinersen in adults with 5q-associated spinal muscular atrophy (SMA) has been demonstrated up to a period of 16 months in relatively large cohorts but whereas patients reach a plateau over time is still to be demonstrated. We investigated the effica...