000281368 001__ 281368
000281368 005__ 20251008102406.0
000281368 0247_ $$2doi$$a10.1186/s13023-025-04009-3
000281368 0247_ $$2pmid$$apmid:40922023
000281368 0247_ $$2pmc$$apmc:PMC12418672
000281368 037__ $$aDZNE-2025-01115
000281368 041__ $$aEnglish
000281368 082__ $$a610
000281368 1001_ $$aWurster, Claudia D$$b0
000281368 245__ $$aRespiratory function in 192 adult patients with spinal muscular atrophy (SMA) treated with nusinersen - a multicenter observational study.
000281368 260__ $$aLondon$$bBioMed Central$$c2025
000281368 3367_ $$2DRIVER$$aarticle
000281368 3367_ $$2DataCite$$aOutput Types/Journal article
000281368 3367_ $$0PUB:(DE-HGF)16$$2PUB:(DE-HGF)$$aJournal Article$$bjournal$$mjournal$$s1759836491_17319
000281368 3367_ $$2BibTeX$$aARTICLE
000281368 3367_ $$2ORCID$$aJOURNAL_ARTICLE
000281368 3367_ $$00$$2EndNote$$aJournal Article
000281368 520__ $$aNatural history data show that respiratory function is impaired in SMA patients. Observational studies have shown stabilization of respiratory function in adult SMA patients treated with nusinersen. However, long-term studies investigating the effect of nusinersen on respiratory function in adult SMA patients are rare.We examined respiratory function using forced vital capacity of predicted normal (FVC%), FVC in liters, capacity per second (FEV1) and peak expiratory flow (PEF) in 192 adult SMA patients treated with nusinersen for a median of 3.2 years (IQR: 2.1–4.0, range: 0.2–5.2). Changes in spirometric parameters were analyzed using individual linear regression models separate in each patient to estimate the slope. Additionally, three multivariable models were performed to assess the effect of age, sex, treatment duration, baseline FVC% and each one of the variables of interest (1) SMA type, (2) ambulation status, (3) spondylodesis on follow-up FVC%. Associations between respiratory parameters and motor function (HFMSE) were investigated via Scatter plots and Spearman’s rank correlation.Spirometric parameters remained stable during treatment (median annual rate of change of FVC% 0.17% (p = 0.40), FVC in liters -0.002 (p = 0.59), FEV1 -0.014 l (p = 0.06) and PEF 0.025 l/s (p = 0.65)). In all multivariable models, age, sex, treatment duration, SMA type, ambulation status, and spondylodesis showed no significant association with follow-up FVC%. No significant correlations were observed between respiratory and motor function.Respiratory parameters remained stable during treatment with nusinersen in adult SMA patients over several years.Not applicable.
000281368 536__ $$0G:(DE-HGF)POF4-353$$a353 - Clinical and Health Care Research (POF4-353)$$cPOF4-353$$fPOF IV$$x0
000281368 588__ $$aDataset connected to CrossRef, PubMed, , Journals: pub.dzne.de
000281368 650_7 $$2Other$$aAdult patients
000281368 650_7 $$2Other$$aForced vital capacity (FVC)
000281368 650_7 $$2Other$$aNusinersen
000281368 650_7 $$2Other$$aRespiratory function
000281368 650_7 $$2Other$$aSpinal muscular atrophy (SMA)
000281368 7001_ $$aStolte, Benjamin$$b1
000281368 7001_ $$aKessler, Tobias$$b2
000281368 7001_ $$aFreigang, Maren$$b3
000281368 7001_ $$aBjelica, Bogdan$$b4
000281368 7001_ $$aIlse, Benjamin$$b5
000281368 7001_ $$aKoch, Jan C$$b6
000281368 7001_ $$aCordts, Isabell$$b7
000281368 7001_ $$aMensch, Alexander$$b8
000281368 7001_ $$aZeller, Daniel$$b9
000281368 7001_ $$aUzelac, Zeljko$$b10
000281368 7001_ $$aSam, Georges$$b11
000281368 7001_ $$aLapp, Hanna Sophie$$b12
000281368 7001_ $$aWohnrade, Camilla$$b13
000281368 7001_ $$aRödiger, Annekathrin$$b14
000281368 7001_ $$aMuhandes, Mohamad Tareq$$b15
000281368 7001_ $$aSchneider, Ilka$$b16
000281368 7001_ $$aBellut, Julia$$b17
000281368 7001_ $$aNentwich, Julia$$b18
000281368 7001_ $$0P:(DE-2719)9001951$$aDorst, Johannes$$b19$$udzne
000281368 7001_ $$0P:(DE-2719)9002279$$aSchuster, Joachim$$b20$$udzne
000281368 7001_ $$aSchreiber-Katz, Olivia$$b21
000281368 7001_ $$aOsmanovic, Alma$$b22
000281368 7001_ $$aTotzeck, Andreas$$b23
000281368 7001_ $$aThimm, Andreas$$b24
000281368 7001_ $$aSteinbach, Robert$$b25
000281368 7001_ $$aGrosskreutz, Julian$$b26
000281368 7001_ $$aKleinschnitz, Christoph$$b27
000281368 7001_ $$0P:(DE-2719)2812633$$aLudolph, Albert C$$b28$$udzne
000281368 7001_ $$aDeschauer, Marcus$$b29
000281368 7001_ $$aKirschner, Janbernd$$b30
000281368 7001_ $$aDreyhaupt, Jens$$b31
000281368 7001_ $$aWollinsky, Kurt$$b32
000281368 7001_ $$aPetri, Susanne$$b33
000281368 7001_ $$aWeiler, Markus$$b34
000281368 7001_ $$aHagenacker, Tim$$b35
000281368 7001_ $$0P:(DE-2719)2811849$$aGünther, René$$b36$$eLast author$$udzne
000281368 773__ $$0PERI:(DE-600)2225857-7$$a10.1186/s13023-025-04009-3$$gVol. 20, no. 1, p. 476$$n1$$p476$$tOrphanet journal of rare diseases$$v20$$x1750-1172$$y2025
000281368 8564_ $$uhttps://pub.dzne.de/record/281368/files/DZNE-2025-01115.pdf$$yOpenAccess
000281368 8564_ $$uhttps://pub.dzne.de/record/281368/files/DZNE-2025-01115.pdf?subformat=pdfa$$xpdfa$$yOpenAccess
000281368 909CO $$ooai:pub.dzne.de:281368$$popenaire$$popen_access$$pVDB$$pdriver$$pdnbdelivery
000281368 9101_ $$0I:(DE-588)1065079516$$6P:(DE-2719)9001951$$aDeutsches Zentrum für Neurodegenerative Erkrankungen$$b19$$kDZNE
000281368 9101_ $$0I:(DE-588)1065079516$$6P:(DE-2719)9002279$$aDeutsches Zentrum für Neurodegenerative Erkrankungen$$b20$$kDZNE
000281368 9101_ $$0I:(DE-588)1065079516$$6P:(DE-2719)2812633$$aDeutsches Zentrum für Neurodegenerative Erkrankungen$$b28$$kDZNE
000281368 9101_ $$0I:(DE-588)1065079516$$6P:(DE-2719)2811849$$aDeutsches Zentrum für Neurodegenerative Erkrankungen$$b36$$kDZNE
000281368 9131_ $$0G:(DE-HGF)POF4-353$$1G:(DE-HGF)POF4-350$$2G:(DE-HGF)POF4-300$$3G:(DE-HGF)POF4$$4G:(DE-HGF)POF$$aDE-HGF$$bGesundheit$$lNeurodegenerative Diseases$$vClinical and Health Care Research$$x0
000281368 9141_ $$y2025
000281368 915__ $$0StatID:(DE-HGF)0200$$2StatID$$aDBCoverage$$bSCOPUS$$d2024-12-11
000281368 915__ $$0StatID:(DE-HGF)0160$$2StatID$$aDBCoverage$$bEssential Science Indicators$$d2024-12-11
000281368 915__ $$0StatID:(DE-HGF)1050$$2StatID$$aDBCoverage$$bBIOSIS Previews$$d2024-12-11
000281368 915__ $$0LIC:(DE-HGF)CCBY4$$2HGFVOC$$aCreative Commons Attribution CC BY 4.0
000281368 915__ $$0StatID:(DE-HGF)0600$$2StatID$$aDBCoverage$$bEbsco Academic Search$$d2024-12-11
000281368 915__ $$0StatID:(DE-HGF)0100$$2StatID$$aJCR$$bORPHANET J RARE DIS : 2022$$d2024-12-11
000281368 915__ $$0StatID:(DE-HGF)0501$$2StatID$$aDBCoverage$$bDOAJ Seal$$d2024-04-10T15:44:07Z
000281368 915__ $$0StatID:(DE-HGF)0500$$2StatID$$aDBCoverage$$bDOAJ$$d2024-04-10T15:44:07Z
000281368 915__ $$0StatID:(DE-HGF)0113$$2StatID$$aWoS$$bScience Citation Index Expanded$$d2024-12-11
000281368 915__ $$0StatID:(DE-HGF)0700$$2StatID$$aFees$$d2024-12-11
000281368 915__ $$0StatID:(DE-HGF)0150$$2StatID$$aDBCoverage$$bWeb of Science Core Collection$$d2024-12-11
000281368 915__ $$0StatID:(DE-HGF)9900$$2StatID$$aIF < 5$$d2024-12-11
000281368 915__ $$0StatID:(DE-HGF)0510$$2StatID$$aOpenAccess
000281368 915__ $$0StatID:(DE-HGF)0030$$2StatID$$aPeer Review$$bASC$$d2024-12-11
000281368 915__ $$0StatID:(DE-HGF)0561$$2StatID$$aArticle Processing Charges$$d2024-12-11
000281368 915__ $$0StatID:(DE-HGF)1120$$2StatID$$aDBCoverage$$bBIOSIS Reviews Reports And Meetings$$d2024-12-11
000281368 915__ $$0StatID:(DE-HGF)0300$$2StatID$$aDBCoverage$$bMedline$$d2024-12-11
000281368 915__ $$0StatID:(DE-HGF)0199$$2StatID$$aDBCoverage$$bClarivate Analytics Master Journal List$$d2024-12-11
000281368 9201_ $$0I:(DE-2719)1710012$$kAG Falkenburger$$lTranslational Parkinson Research$$x0
000281368 9201_ $$0I:(DE-2719)5000077$$kClinical Study Center (Ulm)$$lClinical Study Center (Ulm)$$x1
000281368 980__ $$ajournal
000281368 980__ $$aVDB
000281368 980__ $$aUNRESTRICTED
000281368 980__ $$aI:(DE-2719)1710012
000281368 980__ $$aI:(DE-2719)5000077
000281368 9801_ $$aFullTexts