Nusinersen treatment significantly improves hand grip strength, hand motor function and MRC sum scores in adult patients with spinal muscular atrophy types 3 and 4
Author:
Funder
Biogen
Publisher
Springer Science and Business Media LLC
Subject
Neurology (clinical),Neurology
Link
https://link.springer.com/content/pdf/10.1007/s00415-020-10223-9.pdf
Reference37 articles.
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3. Alías L, Bernal S, Fuentes-Prior P, Barceló MJ, Also E, Martínez-Hernández R et al (2009) Mutation update of spinal muscular atrophy in Spain: Molecular characterization of 745 unrelated patients and identification of four novel mutations in the SMN1 gene. Hum Genet 125(1):29–39
4. Chaytow H, Huang Y, Gillingwater TH, Faller KME (2018) The role of survival motor neuron protein (SMN) in protein homeostasis. Cell Mol Life Sci 75:3877–3894
5. Lorson CL, Androphy EJ (2000) An exonic enhancer is required for inclusion of an essential exon in the SMA-determining gene SMN. Hum Mol Genet 9(2):259–266
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