Type 1 spinal muscular atrophy treated with nusinersen in Norway, a five-year follow-up
Our findings support previously reported results on efficacy and safety of nusinersen. All patients have shown improvement in motor function. The need of respiratory and nutritional support did not improve.
Authors
- Kristin Ørstavik (ORCID: https://orcid.org/0000-0001-7900-7093)
- Magnhild Rasmussen (ORCID: https://orcid.org/0000-0002-2277-8425)
- Sean Wallace (ORCID: https://orcid.org/0000-0001-8520-1052)
- Milada Hagen (ORCID: https://orcid.org/0000-0001-8947-8649)
- Marie Elizabeth Holtebekk
- Henrik Zetterberg (ORCID: https://orcid.org/0000-0003-3930-4354)
- Anette Ramm‐Pettersen
- Merete Wik-Klokk
Institutions
- Oslo University Hospital (NO)
- OsloMet – Oslo Metropolitan University (NO)
- University of Oslo (NO)
- Sahlgrenska University Hospital (SE)
- Metropolitan University (BD)
- University College London (GB)
- Universidad Metropolitana (PR)
Publication Details
- Journal
- European Journal of Paediatric Neurology
- Published
- 2024-10-10
- DOI
- https://doi.org/10.1016/j.ejpn.2024.09.009
- Citations
- 2
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.88
Funders
- Akershus Universitetssykehus