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Assessment of Bulbar Function in Adult Patients with 5q-SMA Type 2 and 3 under Treatment with Nusinersen
The antisense oligonucleotide nusinersen has been shown to improve trunk and limb motor function in patients with spinal muscular atrophy (SMA). Bulbar dysfunction, which is regularly present in SMA, is not captured by standard motor scores, and validated measurement instruments to assess it have no...
Autores principales: | Brakemeier, Svenja, Stolte, Benjamin, Thimm, Andreas, Kizina, Kathrin, Totzeck, Andreas, Munoz-Rosales, Juan, Kleinschnitz, Christoph, Hagenacker, Tim |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8470195/ https://www.ncbi.nlm.nih.gov/pubmed/34573264 http://dx.doi.org/10.3390/brainsci11091244 |
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