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Abstract #0585

Quantitative muscle MRI in monitoring disease progression and nusinersen treatment effects in spinal muscular atrophy

Louise Otto1, Martijn Froeling2, Ruben van Eijk1,3, Renske Wadman1, Inge Cuppen4, Danny van der Woude5, Bart Bartels5, Fay-Lynn Asselman1, Jeroen Hendrikse2, and Ludo van der Pol1
1Department of Neurology, UMC Utrecht Brain Center, University Medical Center, Utrecht, Utrecht, Netherlands, 2Department of Radiology, University Medical Center Utrecht, the Netherlands, Utrecht, Netherlands, 3Biostatistics & Research Support, Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht, Netherlands, 4Department of Neurology and Child Neurology, UMC Utrecht Brain Center, University Medical Center, Utrecht, Utrecht, Netherlands, 5Department of Child Development and Exercise Center, University Medical Center Utrecht, the Netherlands, Utrecht, Netherlands

Quantitative MRI of muscles allows to measure disease progression or to assess therapeutic effects in neuromuscular diseases. We executed two studies on patients with spinal muscular atrophy, treated and untreated, with a protocol consisting of DIXON, T2 mapping and DTI on a 3T MR scanner. In treatment-naïve adult patients we demonstrated that qMRI was able to measure subclinical disease progression. In young children with SMA, quantitative MR parameters of the DIXON and DTI sequence showed ongoing fatty infiltration and normalization of thigh muscle microstructure during the first year of nusinersen treatment.

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