Motor neuron disease

A prospective natural history study of type 1 spinal muscular atrophy

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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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Author information

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.

Correspondence to Enrico Bertini.

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The authors declare no competing financial interests.

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