SMA valiant trial: A prospective, double-blind, placebo-controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy-Reference-Cited by-同舟云学术

SMA valiant trial: A prospective, double-blind, placebo-controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy

Published:2013-06-13 Issue:2 Volume:49 Page:187-192
ISSN:0148-639X
Container-title:Muscle & Nerve
language:en
Short-container-title:Muscle Nerve

Author:

Kissel John T.¹²,Elsheikh Bakri¹,King Wendy M.¹,Freimer Miriam¹,Scott Charles B.³,Kolb Stephen J.¹,Reyna Sandra P.⁴⁵,Crawford Thomas O.⁶⁷,Simard Louise R.⁸,Krosschell Kristin J.⁹,Acsadi Gyula¹⁰¹¹,Schroth Mary K.¹²,D'Anjou Guy¹³,LaSalle Bernard¹⁴,Prior Thomas W.¹⁵,Sorenson Susan¹⁶,Maczulski Jo Anne¹⁷,Swoboda Kathryn J.⁴⁵,

Affiliation:

1. Department of Neurology, Division of Neuromuscular Medicine; Ohio State University, Wexner Medical Center; 395 West 12th Avenue Columbus Ohio 43210 USA

2. Department of Pediatrics; Ohio State University; Columbus Ohio USA

3. CBS Squared, Inc.; Fort Washington; Pennsylvania USA

4. Department of Neurology; University of Utah School of Medicine; Salt Lake City Utah USA

5. Department of Pediatrics; University of Utah School of Medicine; Salt Lake City Utah USA

6. Department of Neurology; Johns Hopkins University School of Medicine; Baltimore Maryland USA

7. Department of Pediatrics; Johns Hopkins University School of Medicine; Baltimore Maryland USA

8. Department of Biochemistry and Medical Genetics; University of Manitoba; Winnipeg Manitoba Canada

9. Department of Physical Therapy and Human Movement Sciences; Feinberg School of Medicine; Northwestern University; Chicago Illinois USA

10. Department of Neurology; University of Connecticut School of Medicine; Hartford Connecticut USA

11. Department of Pediatrics; University of Connecticut School of Medicine; Hartford Connecticut USA

12. Department of Pediatrics; University of Wisconsin School of Medicine; Madison Wisconsin USA

13. Division of Pediatric Neurology; Hôpital Sainte-Justine Montréal; Montréal Québec Canada

14. Department of Biomedical Informatics; University of Utah School of Medicine; Salt Lake City Utah USA

15. Department of Molecular Pathology; Ohio State University, Wexner Medical Center; Columbus Ohio USA

16. Intermountain Healthcare at Primary Children's Medical Center; Salt Lake City Utah USA

17. Pediatric Occupational Therapy Services; Chicago Illinois USA

Funder

Families of Spinal Muscular Atrophy and also by grants from the Center for Clinical and Translational Sciences, University of Utah

Center for Clinical and Translational Sciences, Ohio State University

Publisher

Wiley

Subject

Physiology (medical),Cellular and Molecular Neuroscience,Clinical Neurology,Physiology

Link

http://onlinelibrary.wiley.com/wol1/doi/10.1002/mus.23904/fullpdf

Reference34 articles.

1. Spinal muscular atrophy: a timely review;Kolb;Arch Neurol,2011

2. Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick?;Burghes;Nat Rev Neurosci,2009

3. Spinal muscular atrophy: mechanisms and therapeutic strategies;Lorson;Hum Mol Genet,2010

4. Quantitative analyses of SMN1 and SMN2 based on real-time lightcycler PCR: fast and highly reliable carrier testing and prediction of severity of spinal muscular atrophy;Feldkotter;Am J Hum Genet,2002

5. Correlation between severity and SMN protein level in spinal muscular atrophy;Lefebvre;Nat Genet,1997

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