Nusinersen in adults with 5q spinal muscular atrophy: a non-interventional, multicentre, observational cohort study
The Lancet Neurology
Fecha de publicación: April 2020
Autores: Tim Hagenacker, MD, Claudia D Wurster, MD, René Günther, MD. Olivia Schreiber-Katz, MD, Alma Osmanovic, MD
Prof Susanne Petri, MD, et al.
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.