Longitudinal prospective cohort study to assess peripheral motor function with extensive electrophysiological techniques in patients with Spinal Muscular Atrophy (SMA): the SMA Motor Map protocol

BACKGROUND: Hereditary spinal muscular atrophy (SMA) is a motor neuron disorder with a wide range in severity in children and adults. Two therapies that alter splicing of the Survival Motor Neuron 2 (SMN2) gene, i.e. nusinersen and risdiplam, improve motor function in SMA, but treatment effects vary...

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Detalles Bibliográficos
Autores principales: Ros, Leandra A. A., Goedee, H. Stephan, Franssen, Hessel, Asselman, Fay-Lynn, Bartels, Bart, Cuppen, Inge, van Eijk, Ruben P. A., Sleutjes, Boudewijn T. H. M., van der Pol, W. Ludo, Wadman, Renske I.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Study Protocol
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10124000/
https://www.ncbi.nlm.nih.gov/pubmed/37095427
http://dx.doi.org/10.1186/s12883-023-03207-5

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