Respiratory function in 192 adult patients with spinal muscular atrophy (SMA) treated with nusinersen - a multicenter observational study.

IF 3.5 2区医学 Q2 GENETICS & HEREDITY

Orphanet Journal of Rare Diseases Pub Date : 2025-09-08 DOI:10.1186/s13023-025-04009-3

Claudia D Wurster, Benjamin Stolte, Tobias Kessler, Maren Freigang, Bogdan Bjelica, Benjamin Ilse, Jan C Koch, Isabell Cordts, Alexander Mensch, Daniel Zeller, Zeljko Uzelac, Georges Sam, Hanna Sophie Lapp, Camilla Wohnrade, Annekathrin Rödiger, Mohamad Tareq Muhandes, Ilka Schneider, Julia Bellut, Julia Nentwich, Johannes Dorst, Joachim Schuster, Olivia Schreiber-Katz, Alma Osmanovic, Andreas Totzeck, Andreas Thimm, Robert Steinbach, Julian Grosskreutz, Christoph Kleinschnitz, Albert C Ludolph, Marcus Deschauer, Janbernd Kirschner, Jens Dreyhaupt, Kurt Wollinsky, Susanne Petri, Markus Weiler, Tim Hagenacker, René Günther

引用次数: 0

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nusinersen治疗192例成年脊髓性肌萎缩症（SMA）患者的呼吸功能——一项多中心观察性研究

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来源期刊

Orphanet Journal of Rare Diseases 医学-医学：研究与实验

CiteScore

6.30

自引率

8.10%

发文量

418

审稿时长

4-8 weeks

期刊介绍： Orphanet Journal of Rare Diseases is an open access, peer-reviewed journal that encompasses all aspects of rare diseases and orphan drugs. The journal publishes high-quality reviews on specific rare diseases. In addition, the journal may consider articles on clinical trial outcome reports, either positive or negative, and articles on public health issues in the field of rare diseases and orphan drugs. The journal does not accept case reports.