Longitudinal natural history of type I spinal muscular atrophy: a critical review | Orphanet Journal of Rare Diseases | Full Text

Longitudinal natural history of type I spinal muscular atrophy: a critical review | Orphanet Journal of Rare Diseases | Full Text

Longitudinal natural history of type I spinal muscular atrophy: a critical review

The advent of new therapies in spinal muscular atrophy (SMA) has highlighted the need to have natural history data for comparison. Natural history studies using structured assessments in type I however are ver...

Authors:Eugenio Mercuri, Simona Lucibello, Marco Perulli, Giorgia Coratti, Roberto de Sanctis, Maria Carmela Pera, Marika Pane, Jacqueline Montes, Darryl C. de Vivo, Basil T. Darras, Stephen J. Kolb and Richard S. Finkel

Citation:Orphanet Journal of Rare Diseases 2020 15:84

Content type:Review

Published on: 5 April 2020

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