Spinal muscular atrophy type 3 and treatment adherence in a changing therapeutic landscape: a national-center experience.
Nusinersen had a positive treatment effect despite missed doses during the COVID-19 pandemic. Although the most significant improvements can be expected in patients treated the earliest, nusinersen treatment can be beneficial even in long-standing SMA.
Authors
- Hrvoje Bilić (ORCID: https://orcid.org/0000-0003-2619-6390)
- Mirea Hančević (ORCID: https://orcid.org/0000-0002-7558-1749)
- Barbara Sitaš
- Ervina Bilić (ORCID: https://orcid.org/0000-0003-4466-6528)
- Jakob Nemir (ORCID: https://orcid.org/0000-0002-7250-3016)
- Katarina Bilić
Institutions
- University Hospital Centre Zagreb (HR)
Publication Details
- Journal
- PubMed
- Published
- 2025-02-28
- DOI
- https://doi.org/10.3325/cmj.2025.66.20
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00