A prospective natural history study of type 1 spinal muscular atrophy

Eugenio Maria Mercuri, Enrico Silvio Bertini, Enrico Bertini

Risultato della ricerca: Contributo in rivistaArticolo in rivista

4 Citazioni (Scopus)

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.
Lingua originaleEnglish
pagine (da-a)197-198
Numero di pagine2
RivistaNATURE REVIEWS. NEUROLOGY
Volume14
DOI
Stato di pubblicazionePubblicato - 2018

Keywords

  • Cellular and Molecular Neuroscience
  • Neurology (clinical)

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