Tim Hagenacker, Claudia D Wurster, René Günther, Olivia Schreiber-Katz, Alma Osmanovic, Susanne Petri, Markus Weiler, Andreas Ziegler, Josua Kuttler, Jan C Koch, Ilka Schneider, Gilbert Wunderlich, Natalie Schloss, Helmar C Lehmann, Isabell Cordts, Marcus Deschauer, Paul Lingor, Christoph Kamm, Benjamin Stolte, Lena Pietruck, Andreas Totzeck, Kathrin Kizina, Christoph Mönninghoff, Otgonzul von Velsen, Claudia Ose, Heinz Reichmann, Michael Forsting, Astrid Pechmann, Janbernd Kirschner, Albert C Ludolph, Andreas Hermann, Christoph Kleinschnitz
BACKGROUND: Nusinersen is approved for the treatment of 5q spinal muscular atrophy of all types and stages in patients of all ages. Although clinical trials have shown improvements in motor function in infants and children treated with the drug, data for adults are scarce. We aimed to assess the safety and efficacy of nusinersen in adults with 5q spinal muscular atrophy. METHODS: We did an observational cohort study at ten academic clinical sites in Germany. Patients with genetically confirmed 5q spinal muscular atrophy (age 16-65 years) with a homozygous deletion of exons 7, 8, or both, or with compound heterozygous mutations were eligible for inclusion and received nusinersen treatment in accordance with the label for a minimum treatment time of 6 months to a follow-up of up to 14 months...
April 2020: Lancet Neurology