Spinal Muscular Atrophy Biomarker Measurements from Blood Samples in a Clinical Trial of Valproic Acid in Ambulatory Adults

Background: Clinical trials of therapies for spinal muscular atrophy (SMA) that are designed to increase the expression the SMN protein ideally include careful assessment of relevant SMN biomarkers. Objective: In the SMA VALIANT trial, a recent double-blind placebo-controlled crossover study of valp...

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Detalles Bibliográficos
Autores principales: Renusch, Samantha R., Harshman, Sean, Pi, Hongyang, Workman, Eileen, Wehr, Allison, Li, Xiaobai, Prior, Thomas W., Elsheikh, Bakri H., Swoboda, Kathryn J., Simard, Louise R., Kissel, John T., Battle, Daniel, Parthun, Mark R., Freitas, Michael A., Kolb, Stephen J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2015
Materias:
Research Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5271431/
https://www.ncbi.nlm.nih.gov/pubmed/27858735
http://dx.doi.org/10.3233/JND-150081

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5271431/
https://www.ncbi.nlm.nih.gov/pubmed/27858735
http://dx.doi.org/10.3233/JND-150081

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