Monitoring Nusinersen Treatment Effects in Children with Spinal Muscular Atrophy with Quantitative Muscle MRI

Background: Spinal muscular atrophy (SMA) is caused by deficiency of survival motor neuron (SMN) protein. Intrathecal nusinersen treatment increases SMN protein in motor neurons and has been shown to improve motor function in symptomatic children with SMA. Objective: We used quantitative MRI to gain...

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Veröffentlicht in:	Journal of neuromuscular diseases 2024-01, Vol.11 (1), p.91-101
Hauptverfasser:	Otto, Louise A.M., Froeling, M., van Eijk, Ruben P.A., Wadman, Renske I., Cuppen, Inge, van der Woude, Danny R., Bartels, Bart, Asselman, Fay-Lynn, Hendrikse, Jeroen, van der Pol, W. Ludo
Format:	Artikel
Sprache:	eng
Schlagworte:	Biomarkers Children Magnetic resonance imaging Motor neurons Muscle strength Muscles Research Report SMN protein Spinal muscular atrophy
Online-Zugang:	Volltext
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