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.
Bertini, E. S., Mercuri, E. M., A prospective natural history study of type 1 spinal muscular atrophy, <<NATURE REVIEWS. NEUROLOGY>>, 2018; 14 (4): 197-198. [doi:10.1038/nrneurol.2017.189] [http://hdl.handle.net/10807/122553]
A prospective natural history study of type 1 spinal muscular atrophy
Bertini, Enrico Silvio;Mercuri, Eugenio Maria
2018
Abstract
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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