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Volume 12, Issue 5, Pages (May 2013)

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Presentation on theme: "Volume 12, Issue 5, Pages (May 2013)"— Presentation transcript:

1 Volume 12, Issue 5, Pages 435-442 (May 2013)
An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study  Dr Timothy M Miller, MD, Prof Alan Pestronk, MD, William David, MD, Prof Jeffrey Rothstein, MD, Ericka Simpson, MD, Prof Stanley H Appel, MD, Patricia L Andres, MS, Katy Mahoney, BA, Peggy Allred, DPT, Katie Alexander, BA, Lyle W Ostrow, MD, Prof David Schoenfeld, PhD, Eric A Macklin, PhD, Daniel A Norris, PhD, Georgios Manousakis, MD, Matthew Crisp, BS, Richard Smith, MD, C Frank Bennett, PhD, Kathie M Bishop, PhD, Prof Merit E Cudkowicz, MD  The Lancet Neurology  Volume 12, Issue 5, Pages (May 2013) DOI: /S (13) Copyright © 2013 Elsevier Ltd Terms and Conditions

2 Figure 3 SOD1 protein concentrations in CSF of patients enrolled in more than one cohort Measured by ELISA. SOD1 mutation and cohort number are shown for each patient. *Placebo group for that cohort. The Lancet Neurology  , DOI: ( /S (13) ) Copyright © 2013 Elsevier Ltd Terms and Conditions


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