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

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

Abstract 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 eff...

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Bibliographic Details
Main Authors: Leandra A. A. Ros, H. Stephan Goedee, Hessel Franssen, Fay-Lynn Asselman, Bart Bartels, Inge Cuppen, Ruben P. A. van Eijk, Boudewijn T. H. M. Sleutjes, W. Ludo van der Pol, Renske I. Wadman
Format: Article
Language:English
Published: BMC 2023-04-01
Series:BMC Neurology
Subjects:
Spinal muscular atrophy
Nusinersen
Risdiplam
Electrophysiological techniques
Motor unit
Online Access:https://doi.org/10.1186/s12883-023-03207-5

Internet

https://doi.org/10.1186/s12883-023-03207-5

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