Literature DB >> 16775228

Valproate may improve strength and function in patients with type III/IV spinal muscle atrophy.

Conrad C Weihl1, Anne M Connolly, Alan Pestronk.   

Abstract

Spinal muscular atrophy (SMA) is a recessively inherited motor neuron disease caused by deficient survival motor neuron (SMN) protein. Valproate increases SMN protein in vitro by increasing transcription of SMN2 genes. The authors treated seven adult patients with SMA type III/IV with valproate for a mean duration of 8 months. The treated patients with SMA had increased quantitative muscle strength and subjective function. Further trials of valproate treatment for SMA type III/IV are warranted.

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Year:  2006        PMID： 16775228     DOI： 10.1212/01.wnl.0000231139.26253.d0

Source DB:  PubMed          Journal:  Neurology        ISSN： 0028-3878            Impact factor:   9.910


  43 in total

1.  Population pharmacokinetics of valproic acid in pediatric patients with epilepsy: considerations for dosing spinal muscular atrophy patients.

Authors:  Jason H Williams; Bhuvaneswari Jayaraman; Kathryn J Swoboda; Jeffrey S Barrett
Journal:  J Clin Pharmacol       Date:  2011-12-13       Impact factor: 3.126

Review 2.  Applicability of histone deacetylase inhibition for the treatment of spinal muscular atrophy.

Authors:  Sebastian Lunke; Assam El-Osta
Journal:  Neurotherapeutics       Date:  2013-10       Impact factor: 7.620

3.  Development of histone deacetylase inhibitors as therapeutics for neurological disease.

Authors:  Joel M Gottesfeld; Massimo Pandolfo
Journal:  Future Neurol       Date:  2009-11-01

4.  Spinal muscular atrophy: advances in research and consensus on care of patients.

Authors:  Ching H Wang; Mitchell R Lunn
Journal:  Curr Treat Options Neurol       Date:  2008-11       Impact factor: 3.598

5.  SMN transcript levels in leukocytes of SMA patients determined by absolute real-time PCR.

Authors:  Francesco Danilo Tiziano; Anna Maria Pinto; Stefania Fiori; Rosa Lomastro; Sonia Messina; Claudio Bruno; Antonella Pini; Marika Pane; Adele D'Amico; Alessandro Ghezzo; Enrico Bertini; Eugenio Mercuri; Giovanni Neri; Christina Brahe
Journal:  Eur J Hum Genet       Date:  2010-01       Impact factor: 4.246

Review 6.  Targeting RNA-splicing for SMA treatment.

Authors:  Jianhua Zhou; Xuexiu Zheng; Haihong Shen
Journal:  Mol Cells       Date:  2012-02-28       Impact factor: 5.034

7.  Drug treatment for spinal muscular atrophy types II and III.

Authors:  Renske I Wadman; W Ludo van der Pol; Wendy Mj Bosboom; Fay-Lynn Asselman; Leonard H van den Berg; Susan T Iannaccone; Alexander Fje Vrancken
Journal:  Cochrane Database Syst Rev       Date:  2020-01-06

Review 8.  Multiple roles of HDAC inhibition in neurodegenerative conditions.

Authors:  De-Maw Chuang; Yan Leng; Zoya Marinova; Hyeon-Ju Kim; Chi-Tso Chiu
Journal:  Trends Neurosci       Date:  2009-09-21       Impact factor: 13.837

9.  SMA CARNI-VAL trial part I: double-blind, randomized, placebo-controlled trial of L-carnitine and valproic acid in spinal muscular atrophy.

Authors:  Kathryn J Swoboda; Charles B Scott; Thomas O Crawford; Louise R Simard; Sandra P Reyna; Kristin J Krosschell; Gyula Acsadi; Bakri Elsheik; Mary K Schroth; Guy D'Anjou; Bernard LaSalle; Thomas W Prior; Susan L Sorenson; Jo Anne Maczulski; Mark B Bromberg; Gary M Chan; John T Kissel
Journal:  PLoS One       Date:  2010-08-19       Impact factor: 3.240

10.  Severe SMA mice show organ impairment that cannot be rescued by therapy with the HDACi JNJ-26481585.

Authors:  Julia Schreml; Markus Riessland; Mario Paterno; Lutz Garbes; Kristina Roßbach; Bastian Ackermann; Jan Krämer; Eilidh Somers; Simon H Parson; Raoul Heller; Albrecht Berkessel; Anja Sterner-Kock; Brunhilde Wirth
Journal:  Eur J Hum Genet       Date:  2012-10-17       Impact factor: 4.246
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