Real-world data on the effect of long-term treatment with nusinersen over > 4 years in a cohort of Swiss patients with spinal muscular atrophy
Our data confirm sustained gains of motor function in early-treated patients and disease stabilisation in patients in the chronic phase under nusinersen treatment.
Authors
- Andrea Klein (ORCID: https://orcid.org/0000-0003-2733-5911)
- Georg Martin Stettner (ORCID: https://orcid.org/0000-0002-0906-8816)
- Paolo Ripellino (ORCID: https://orcid.org/0000-0002-8662-9656)
- Esther Irene Schwarz (ORCID: https://orcid.org/0000-0001-8840-981X)
- Cornelia Enzmann (ORCID: https://orcid.org/0000-0002-6780-3348)
- Bettina Schreiner (ORCID: https://orcid.org/0000-0002-3830-6991)
- Olivier Scheidegger (ORCID: https://orcid.org/0000-0003-3925-5142)
- Christoph Neuwirth (ORCID: https://orcid.org/0000-0002-1874-8872)
- Elea Galiart
- Leonie Steiner
- Bettina Henzi
- Philip J. Broser (ORCID: https://orcid.org/0000-0003-4477-1432)
- David Jacquier (ORCID: https://orcid.org/0000-0002-6173-2905)
- Andrea Mathis (ORCID: https://orcid.org/0000-0002-4190-0414)
- Anne Tscherter (ORCID: https://orcid.org/0000-0002-6972-482X)
- Gian Paolo Ramelli
Institutions
- University of Bern (CH)
- University of Zurich (CH)
- University of St.Gallen (CH)
- University Hospital of Bern (CH)
- Laboratory for Biomedical Neurosciences (CH)
- Ospedale San Giovanni Bellinzona (CH)
- University Hospital of Zurich (CH)
- Institute of Oncology Research (CH)
- University Children’s Hospital Bern (CH)
- University Children’s Hospital Basel (CH)
- University Children's Hospital Zurich (CH)
- Institute of Social and Preventive Medicine (CH)
- Università della Svizzera italiana (CH)
- University of Lausanne (CH)
Publication Details
- Journal
- Clinical Neurology and Neurosurgery
- Published
- 2025-05-26
- DOI
- https://doi.org/10.1016/j.clineuro.2025.108983
- Citations
- 4
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 6.50
Funders
- Pfizer
- Biogen
- Universität Basel
- University of Bern
- Stiftung für die Erforschung der Muskelkrankheiten
- PTC Therapeutics
- Sarepta Therapeutics
- Inselspital, Universitätsspital Bern
- Centre Hospitalier Universitaire Vaudois
- Universitätsspital Zürich