A Comparative Study of SMN Protein and mRNA in Blood and Fibroblasts in Patients with Spinal Muscular Atrophy and Healthy Controls

BACKGROUND: Clinical trials to test safety and efficacy of drugs for patients with spinal muscular atrophy (SMA) are currently underway. Biomarkers that document treatment-induced effects are needed because disease progression in childhood forms of SMA is slow and clinical outcome measures may lack...

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Vydáno v:PLoS One
Hlavní autoři: Wadman, Renske I., Stam, Marloes, Jansen, Marc D., van der Weegen, Yana, Wijngaarde, Camiel A., Harschnitz, Oliver, Sodaar, Peter, Braun, Kees P. J., Dooijes, Dennis, Lemmink, Henny H., van den Berg, Leonard H., van der Pol, W. Ludo
Médium: Artigo
Jazyk:Inglês
Vydáno: Public Library of Science 2016
Témata:
Research Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5125671/
https://ncbi.nlm.nih.gov/pubmed/27893852
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0167087
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