A prospective natural history study of type 1 spinal muscular atrophy

Enrico Bertini; Eugenio Maria Mercuri

doi:10.1038/nrneurol.2017.189

A prospective natural history study of type 1 spinal muscular atrophy

Enrico Bertini^*
, Eugenio Maria Mercuri

^*Corresponding author

IRCCS Ospedale pediatrico Bambino Gesù - Roma

Research output: Contribution to journal › Article

4 Citations (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.

Original language	English
Pages (from-to)	197-198
Number of pages	2
Journal	Nature Reviews Neurology
Volume	14
Issue number	4
DOIs	https://doi.org/10.1038/nrneurol.2017.189
Publication status	Published - 2018

All Science Journal Classification (ASJC) codes

Clinical Neurology
Cellular and Molecular Neuroscience

Keywords

Cellular and Molecular Neuroscience
Neurology (clinical)

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10.1038/nrneurol.2017.189

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title = "A prospective natural history study of type 1 spinal muscular atrophy",

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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AU - Mercuri, Eugenio Maria

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AB - 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.

KW - Cellular and Molecular Neuroscience

KW - Neurology (clinical)

KW - Cellular and Molecular Neuroscience

KW - Neurology (clinical)

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A prospective natural history study of type 1 spinal muscular atrophy

Abstract

All Science Journal Classification (ASJC) codes

Keywords

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