%0 Journal Article
%A Wurster, Claudia D
%A Stolte, Benjamin
%A Kessler, Tobias
%A Freigang, Maren
%A Bjelica, Bogdan
%A Ilse, Benjamin
%A Koch, Jan C
%A Cordts, Isabell
%A Mensch, Alexander
%A Zeller, Daniel
%A Uzelac, Zeljko
%A Sam, Georges
%A Lapp, Hanna Sophie
%A Wohnrade, Camilla
%A Rödiger, Annekathrin
%A Muhandes, Mohamad Tareq
%A Schneider, Ilka
%A Bellut, Julia
%A Nentwich, Julia
%A Dorst, Johannes
%A Schuster, Joachim
%A Schreiber-Katz, Olivia
%A Osmanovic, Alma
%A Totzeck, Andreas
%A Thimm, Andreas
%A Steinbach, Robert
%A Grosskreutz, Julian
%A Kleinschnitz, Christoph
%A Ludolph, Albert C
%A Deschauer, Marcus
%A Kirschner, Janbernd
%A Dreyhaupt, Jens
%A Wollinsky, Kurt
%A Petri, Susanne
%A Weiler, Markus
%A Hagenacker, Tim
%A Günther, René
%T Respiratory function in 192 adult patients with spinal muscular atrophy (SMA) treated with nusinersen - a multicenter observational study.
%J Orphanet journal of rare diseases
%V 20
%N 1
%@ 1750-1172
%C London
%I BioMed Central
%M DKFZ-2025-01871
%P 476
%D 2025
%X Natural history data show that respiratory function is impaired in SMA patients. Observational studies have shown stabilization of respiratory function in adult SMA patients treated with nusinersen. However, long-term studies investigating the effect of nusinersen on respiratory function in adult SMA patients are rare.We examined respiratory function using forced vital capacity of predicted normal (FVC
%K Adult patients (Other)
%K Forced vital capacity (FVC) (Other)
%K Nusinersen (Other)
%K Respiratory function (Other)
%K Spinal muscular atrophy (SMA) (Other)
%F PUB:(DE-HGF)16
%9 Journal Article
%$ pmid:40922023
%2 pmc:PMC12418672
%R 10.1186/s13023-025-04009-3
%U https://inrepo02.dkfz.de/record/304479