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

Publikation

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

Wissenschaftlicher Artikel/Review - 06.02.2024

Günther René, Wurster Claudia Diana, Brakemeier Svenja, Osmanovic Alma, Schreiber-Katz Olivia, Petri Susanne, Uzelac Zeljko, Hiebeler Miriam, Thiele Simone, Walter M C, Weiler Markus, Kessler Tobias, Freigang Maren, Lapp Hanna Sophie, Cordts Isabell, Lingor Paul, Deschauer Marcus, Hahn Andreas, Martakis Kyriakos, Steinbach Robert, Ilse Benjamin, Roediger Annekathrin, Bellut Julia, Nentwich Julia, Zeller Daniel, Muhandes Mohamad Tareq, Baum Tobias, Koch Jan Christoph, Schrank Bertold, Fischer Sophie, Hermann Andreas, Kamm Christoph, Naegel Steffen, Mensch Alexander, Weber Markus, Neuwirth Christoph, Lehmann Helmar C, Wunderlich Gilbert, Stadler Christian, Tomforde Maike, George Annette, Gross Martin, Pechmann Astrid, Kirschner Janbernd, Türk Matthias, Schimmel Mareike, Bernert Günther, Martin Pascal, Rauscher Christian, Meyer Zu Hörste Gerd, Baum Petra, Löscher Wolfgang, Flotats-Bastardas Marina, Köhler Cornelia, Probst-Schendzielorz Kristina, Goldbach Susanne, Schara-Schmidt Ulrike, Mueller-Felber Wolfgang, Lochmüller Hanns, von Velsen Otgonzul, SMArtCARE study group, Kleinschnitz Christoph, Ludolph Albert C, Hagenacker Tim

Zitation

Günther R, Wurster C, Brakemeier S, Osmanovic A, Schreiber-Katz O, Petri S, Uzelac Z, Hiebeler M, Thiele S, Walter M, Weiler M, Kessler T, Freigang M, Lapp H, Cordts I, Lingor P, Deschauer M, Hahn A, Martakis K, Steinbach R, Ilse B, Roediger A, Bellut J, Nentwich J, Zeller D, Muhandes M, Baum T, Koch J, Schrank B, Fischer S, Hermann A, Kamm C, Naegel S, Mensch A, Weber M, Neuwirth C, Lehmann H, Wunderlich G, Stadler C, Tomforde M, George A, Gross M, Pechmann A, Kirschner J, Türk M, Schimmel M, Bernert G, Martin P, Rauscher C, Meyer Zu Hörste G, Baum P, Löscher W, Flotats-Bastardas M, Köhler C, Probst-Schendzielorz K, Goldbach S, Schara-Schmidt U, Mueller-Felber W, Lochmüller H, von Velsen O, SMArtCARE study group, Kleinschnitz C, Ludolph A, Hagenacker T. Long-term efficacy and safety of nusinersen in adults with 5q spinal muscular atrophy: a prospective European multinational observational study. Lancet Reg Health Eur 2024; 39:100862.

Art

Wissenschaftlicher Artikel/Review (Englisch)

Zeitschrift

Lancet Reg Health Eur 2024; 39

Veröffentlichungsdatum

06.02.2024

eISSN (Online)

2666-7762

Seiten

100862

Kurzbeschreibung/Zielsetzung

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 efficacy and safety of nusinersen in adults with SMA over 38 months, the longest time period to date in a large cohort of patients from multiple clinical sites.