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

BACKGROUND: 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 very limited. We identified and reviewed all the existing longitudinal history dat...

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Spremljeno u:
Bibliografski detalji
Izdano u:Orphanet J Rare Dis
Glavni autori: Mercuri, Eugenio, Lucibello, Simona, Perulli, Marco, Coratti, Giorgia, de Sanctis, Roberto, Pera, Maria Carmela, Pane, Marika, Montes, Jacqueline, de Vivo, Darryl C., Darras, Basil T., Kolb, Stephen J., Finkel, Richard S.
Format: Artigo
Jezik:Inglês
Izdano: BioMed Central 2020
Teme:
Review
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7132885/
https://ncbi.nlm.nih.gov/pubmed/32248834
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-020-01356-1
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