TY  - JOUR
AU  - Wurster, Claudia D
AU  - Stolte, Benjamin
AU  - Kessler, Tobias
AU  - Freigang, Maren
AU  - Bjelica, Bogdan
AU  - Ilse, Benjamin
AU  - Koch, Jan C
AU  - Cordts, Isabell
AU  - Mensch, Alexander
AU  - Zeller, Daniel
AU  - Uzelac, Zeljko
AU  - Sam, Georges
AU  - Lapp, Hanna Sophie
AU  - Wohnrade, Camilla
AU  - Rödiger, Annekathrin
AU  - Muhandes, Mohamad Tareq
AU  - Schneider, Ilka
AU  - Bellut, Julia
AU  - Nentwich, Julia
AU  - Dorst, Johannes
AU  - Schuster, Joachim
AU  - Schreiber-Katz, Olivia
AU  - Osmanovic, Alma
AU  - Totzeck, Andreas
AU  - Thimm, Andreas
AU  - Steinbach, Robert
AU  - Grosskreutz, Julian
AU  - Kleinschnitz, Christoph
AU  - Ludolph, Albert C
AU  - Deschauer, Marcus
AU  - Kirschner, Janbernd
AU  - Dreyhaupt, Jens
AU  - Wollinsky, Kurt
AU  - Petri, Susanne
AU  - Weiler, Markus
AU  - Hagenacker, Tim
AU  - Günther, René
TI  - Respiratory function in 192 adult patients with spinal muscular atrophy (SMA) treated with nusinersen - a multicenter observational study.
JO  - Orphanet journal of rare diseases
VL  - 20
IS  - 1
SN  - 1750-1172
CY  - London
PB  - BioMed Central
M1  - DKFZ-2025-01871
SP  - 476
PY  - 2025
AB  - 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
KW  - Adult patients (Other)
KW  - Forced vital capacity (FVC) (Other)
KW  - Nusinersen (Other)
KW  - Respiratory function (Other)
KW  - Spinal muscular atrophy (SMA) (Other)
LB  - PUB:(DE-HGF)16
C6  - pmid:40922023
C2  - pmc:PMC12418672
DO  - DOI:10.1186/s13023-025-04009-3
UR  - https://inrepo02.dkfz.de/record/304479
ER  -