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Effect of nusinersen on motor, respiratory and bulbar function in early-onset spinal muscular atrophy.
Brain. 2023 Feb 13;146(2):668-677. doi: 10.1093/brain/awac252.
Brain. 2023.
PMID: 35857854
Cathepsin D as biomarker in cerebrospinal fluid of nusinersen-treated patients with spinal muscular atrophy.
Schorling DC, Kölbel H, Hentschel A, Pechmann A, Meyer N, Wirth B, Rombo R; SMArtCARE consortium; Sickmann A, Kirschner J, Schara-Schmidt U, Lochmüller H, Roos A.
Schorling DC, et al.
Eur J Neurol. 2022 Jul;29(7):2084-2096. doi: 10.1111/ene.15331. Epub 2022 May 4.
Eur J Neurol. 2022.
PMID: 35318785
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