Effective drug development for infantile-onset spinal muscular atrophy (SMA) requires a meaningful understanding of disease progression and reliable biomarkers. A new report presents the results of a longitudinal, multicentre, prospective natural history study of SMA, which are critical for the research of future therapies.
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E.B. researched data for and wrote the article. Both E.B. and E.M. carried out discussions of the content and the review/editing of the manuscript.
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Bertini, E., Mercuri, E. A prospective natural history study of type 1 spinal muscular atrophy. Nat Rev Neurol 14, 197–198 (2018). https://doi.org/10.1038/nrneurol.2017.189
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DOI: https://doi.org/10.1038/nrneurol.2017.189
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