Evaluation of SMN protein, transcript, and copy number in the biomarkers for spinal muscular atrophy (BforSMA) clinical study.

Evaluation of SMN protein, transcript, and copy number in the biomarkers for spinal muscular atrophy (BforSMA) clinical study.

<label>BACKGROUND</label>The universal presence of a gene (SMN2) nearly identical to the mutated SMN1 gene responsible for Spinal Muscular Atrophy (SMA) has proved an enticing incentive to therapeutics development. Early disappointments from putative SMN-enhancing agent clinical trials h...

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Bibliographic Details
Main Authors: Thomas O Crawford, Sergey V Paushkin, Dione T Kobayashi, Suzanne J Forrest, Cynthia L Joyce, Richard S Finkel, Petra Kaufmann, Kathryn J Swoboda, Danilo Tiziano, Rosa Lomastro, Rebecca H Li, Felicia L Trachtenberg, Thomas Plasterer, Karen S Chen, Pilot Study of Biomarkers for Spinal Muscular Atrophy Trial Group
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2012-01-01
Series:PLoS ONE
Online Access:http://europepmc.org/articles/PMC3338744?pdf=render

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http://europepmc.org/articles/PMC3338744?pdf=render

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