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...

Source: Orphanet Journal of Rare Diseases - April 5, 2020 Category: Internal Medicine 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 Tags: Review Source Type: research