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

Louise A M Otto,M Froeling,Ruben P A van Eijk,Renske I Wadman,Inge Cuppen,Danny R van der Woude,Bart Bartels,Fay-Lynn Asselman,Jeroen Hendrikse,W Ludo van der Pol

Journal of neuromuscular diseases（2023）

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摘要

Thigh muscles of children with SMA treated with nusinersen showed ongoing fatty infiltration and possible normalization of thigh muscle microstructure during the first year of nusinersen treatment. Quantitative muscle MRI shows potential as biomarker for the effects of SMA treatment strategies.

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