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

ABSTRACT Introduction: An open‐label trial suggested that valproic acid (VPA) improved strength in adults with spinal muscular atrophy (SMA). We report a 12‐month, double‐blind, cross‐over study of VPA in ambulatory SMA adults. Methods: There were 33 subjects, aged 20–55 years, included in this inve...

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Published inMuscle & nerve Vol. 49; no. 2; pp. 187 - 192
Main Authors Kissel, John T., Elsheikh, Bakri, King, Wendy M., Freimer, Miriam, Scott, Charles B., Kolb, Stephen J., Reyna, Sandra P., Crawford, Thomas O., Simard, Louise R., Krosschell, Kristin J., Acsadi, Gyula, Schroth, Mary K., D'Anjou, Guy, LaSalle, Bernard, Prior, Thomas W., Sorenson, Susan, Maczulski, Jo Anne, Swoboda, Kathryn J.
Format Journal Article
LanguageEnglish
Published United States Blackwell Publishing Ltd 01.02.2014
Wiley Subscription Services, Inc
Subjects
Adult
Ambulatory Care
carnitine
Cohort Studies
Cross-Over Studies
Dose-Response Relationship, Drug
Double-Blind Method
Female
Histone Deacetylase Inhibitors - pharmacology
Histone Deacetylase Inhibitors - therapeutic use
Humans
Male
Middle Aged
motor neuron disease
Muscle Contraction - drug effects
Muscle Contraction - physiology
Muscle Strength - drug effects
Muscle Strength - physiology
Muscular Atrophy, Spinal - drug therapy
Muscular Atrophy, Spinal - physiopathology
Muscular system
Prospective Studies
spinal muscular atrophy
Treatment Outcome
valproic acid
Valproic Acid - pharmacology
Valproic Acid - therapeutic use
Online AccessGet full text
ISSN0148-639X
1097-4598
1097-4598
DOI10.1002/mus.23904

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Abstract ABSTRACT Introduction: An open‐label trial suggested that valproic acid (VPA) improved strength in adults with spinal muscular atrophy (SMA). We report a 12‐month, double‐blind, cross‐over study of VPA in ambulatory SMA adults. Methods: There were 33 subjects, aged 20–55 years, included in this investigation. After baseline assessment, subjects were randomized to receive VPA (10–20 mg/kg/day) or placebo. At 6 months, patients were switched to the other group. Assessments were performed at 3, 6, and 12 months. The primary outcome was the 6‐month change in maximum voluntary isometric contraction testing with pulmonary, electrophysiological, and functional secondary outcomes. Results: Thirty subjects completed the study. VPA was well tolerated, and compliance was good. There was no change in primary or secondary outcomes at 6 or 12 months. Conclusions: VPA did not improve strength or function in SMA adults. The outcomes used are feasible and reliable and can be employed in future trials in SMA adults. Muscle Nerve 49: 187–192, 2014
AbstractList Introduction: An open-label trial suggested that valproic acid (VPA) improved strength in adults with spinal muscular atrophy (SMA). We report a 12-month, double-blind, cross-over study of VPA in ambulatory SMA adults. Methods: There were 33 subjects, aged 20-55 years, included in this investigation. After baseline assessment, subjects were randomized to receive VPA (10-20 mg/kg/day) or placebo. At 6 months, patients were switched to the other group. Assessments were performed at 3, 6, and 12 months. The primary outcome was the 6-month change in maximum voluntary isometric contraction testing with pulmonary, electrophysiological, and functional secondary outcomes. Results: Thirty subjects completed the study. VPA was well tolerated, and compliance was good. There was no change in primary or secondary outcomes at 6 or 12 months. Conclusions: VPA did not improve strength or function in SMA adults. The outcomes used are feasible and reliable and can be employed in future trials in SMA adults. Muscle Nerve 49: 187-192, 2014 [PUBLICATION ABSTRACT]
An open-label trial suggested that valproic acid (VPA) improved strength in adults with spinal muscular atrophy (SMA). We report a 12-month, double-blind, cross-over study of VPA in ambulatory SMA adults. There were 33 subjects, aged 20–55 years, included in this investigation. After baseline assessment, subjects were randomized to receive VPA (10–20 mg/kg/day) or placebo. At 6 months, patients were switched to the other group. Assessments were performed at 3, 6, and 12 months. The primary outcome was the 6-month change in maximum voluntary isometric contraction testing with pulmonary, electrophysiological, and functional secondary outcomes. Thirty subjects completed the study. VPA was well tolerated, and compliance was good. There was no change in primary or secondary outcomes at 6 or 12 months. VPA did not improve strength or function in SMA adults. The outcomes used are feasible and reliable and can be employed in future trials in SMA adults.
An open-label trial suggested that valproic acid (VPA) improved strength in adults with spinal muscular atrophy (SMA). We report a 12-month, double-blind, cross-over study of VPA in ambulatory SMA adults.INTRODUCTIONAn open-label trial suggested that valproic acid (VPA) improved strength in adults with spinal muscular atrophy (SMA). We report a 12-month, double-blind, cross-over study of VPA in ambulatory SMA adults.There were 33 subjects, aged 20–55 years, included in this investigation. After baseline assessment, subjects were randomized to receive VPA (10–20 mg/kg/day) or placebo. At 6 months, patients were switched to the other group. Assessments were performed at 3, 6, and 12 months. The primary outcome was the 6-month change in maximum voluntary isometric contraction testing with pulmonary, electrophysiological, and functional secondary outcomes.METHODSThere were 33 subjects, aged 20–55 years, included in this investigation. After baseline assessment, subjects were randomized to receive VPA (10–20 mg/kg/day) or placebo. At 6 months, patients were switched to the other group. Assessments were performed at 3, 6, and 12 months. The primary outcome was the 6-month change in maximum voluntary isometric contraction testing with pulmonary, electrophysiological, and functional secondary outcomes.Thirty subjects completed the study. VPA was well tolerated, and compliance was good. There was no change in primary or secondary outcomes at 6 or 12 months.RESULTSThirty subjects completed the study. VPA was well tolerated, and compliance was good. There was no change in primary or secondary outcomes at 6 or 12 months.VPA did not improve strength or function in SMA adults. The outcomes used are feasible and reliable and can be employed in future trials in SMA adults.CONCLUSIONSVPA did not improve strength or function in SMA adults. The outcomes used are feasible and reliable and can be employed in future trials in SMA adults.
Introduction: An open-label trial suggested that valproic acid (VPA) improved strength in adults with spinal muscular atrophy (SMA). We report a 12-month, double-blind, cross-over study of VPA in ambulatory SMA adults. Methods: There were 33 subjects, aged 20-55 years, included in this investigation. After baseline assessment, subjects were randomized to receive VPA (10-20 mg/kg/day) or placebo. At 6 months, patients were switched to the other group. Assessments were performed at 3, 6, and 12 months. The primary outcome was the 6-month change in maximum voluntary isometric contraction testing with pulmonary, electrophysiological, and functional secondary outcomes. Results: Thirty subjects completed the study. VPA was well tolerated, and compliance was good. There was no change in primary or secondary outcomes at 6 or 12 months. Conclusions: VPA did not improve strength or function in SMA adults. The outcomes used are feasible and reliable and can be employed in future trials in SMA adults. Muscle Nerve 49: 187-192, 2014
ABSTRACT Introduction: An open‐label trial suggested that valproic acid (VPA) improved strength in adults with spinal muscular atrophy (SMA). We report a 12‐month, double‐blind, cross‐over study of VPA in ambulatory SMA adults. Methods: There were 33 subjects, aged 20–55 years, included in this investigation. After baseline assessment, subjects were randomized to receive VPA (10–20 mg/kg/day) or placebo. At 6 months, patients were switched to the other group. Assessments were performed at 3, 6, and 12 months. The primary outcome was the 6‐month change in maximum voluntary isometric contraction testing with pulmonary, electrophysiological, and functional secondary outcomes. Results: Thirty subjects completed the study. VPA was well tolerated, and compliance was good. There was no change in primary or secondary outcomes at 6 or 12 months. Conclusions: VPA did not improve strength or function in SMA adults. The outcomes used are feasible and reliable and can be employed in future trials in SMA adults. Muscle Nerve 49: 187–192, 2014
Introduction : An open‐label trial suggested that valproic acid (VPA) improved strength in adults with spinal muscular atrophy (SMA). We report a 12‐month, double‐blind, cross‐over study of VPA in ambulatory SMA adults. Methods : There were 33 subjects, aged 20–55 years, included in this investigation. After baseline assessment, subjects were randomized to receive VPA (10–20 mg/kg/day) or placebo. At 6 months, patients were switched to the other group. Assessments were performed at 3, 6, and 12 months. The primary outcome was the 6‐month change in maximum voluntary isometric contraction testing with pulmonary, electrophysiological, and functional secondary outcomes. Results : Thirty subjects completed the study. VPA was well tolerated, and compliance was good. There was no change in primary or secondary outcomes at 6 or 12 months. Conclusions : VPA did not improve strength or function in SMA adults. The outcomes used are feasible and reliable and can be employed in future trials in SMA adults. Muscle Nerve 49 : 187–192, 2014
Author Reyna, Sandra P.
Acsadi, Gyula
LaSalle, Bernard
Schroth, Mary K.
Swoboda, Kathryn J.
Prior, Thomas W.
King, Wendy M.
Simard, Louise R.
D'Anjou, Guy
Scott, Charles B.
Elsheikh, Bakri
Sorenson, Susan
Freimer, Miriam
Maczulski, Jo Anne
Kissel, John T.
Krosschell, Kristin J.
Kolb, Stephen J.
Crawford, Thomas O.
AuthorAffiliation 7 Department of Pediatrics, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA
17 Pediatric Occupational Therapy Services, Chicago, Illinois, USA
16 Intermountain Healthcare at Primary Children’s Medical Center, Salt Lake City, Utah, USA
2 Department of Pediatrics, Ohio State University, Columbus, Ohio, USA
5 Department of Pediatrics, University of Utah School of Medicine, Salt Lake City, Utah, USA
13 Division of Pediatric Neurology, Hôpital Sainte-Justine Montréal, Montréal, Québec, Canada
1 Department of Neurology, Division of Neuromuscular Medicine, Ohio State University, Wexner Medical Center, 395 West 12th Avenue, Columbus, Ohio, 43210, USA
4 Department of Neurology, University of Utah School of Medicine, Salt Lake City, Utah, USA
10 Department of Neurology, University of Connecticut School of Medicine, Hartford, Connecticut, USA
14 Department of Biomedical Informatics, University of Utah School of Medicine, Salt Lake City, Utah, USA
6 Department of Neurology, Johns Hopki
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BackLink https://www.ncbi.nlm.nih.gov/pubmed/23681940$$D View this record in MEDLINE/PubMed
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Notes Center for Clinical and Translational Sciences, Ohio State University - No. UL1RR025755
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Families of Spinal Muscular Atrophy and also by grants from the Center for Clinical and Translational Sciences, University of Utah - No. UL1RR025764
J.K. received drugs from Abbott Pharmaceuticals for a clinical trial in SMA, is a paid consultant for Alexion Pharmaceuticals and Cytokinetics, and is funded by the National Institutes of Health (NIH U10 NS77382‐2 for NeuroNEXT). B.E. received drugs from Abbott Pharmaceuticals for a clinical trial in SMA. S.R. has received grants from Families of SMA. K.K has received grant funding from the Families of SMA. G.A. receives funding from NIH/NINDS. B.L. received grants from Families of SMA and the National Center for Research Resources (UL1RR025764 to the University of Utah Center for Clinical and Translational Science). K.S. has contracts with ISIS Pharmaceuticals, Inc., Orphamed, and Biomarin for clinical trials and receives grant support from the NIH (R01‐HD69045 from NICHD and U10 NS077305 from NINDS), the Muscular Dystrophy Association, and the Alternating Hemiplegia of Childhood Foundation. The remaining authors have no disclosures to report.
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References Lorson CL, Rindt H, Shababi M. Spinal muscular atrophy: mechanisms and therapeutic strategies. Hum Mol Genet 2010;19:R111-R118.
Tsai LK, Yang CC, Hwu WL, Li H. Valproic acid treatment in six patients with spinal muscular atrophy. Eur J Neurol 2007;14:e8-e9.
Feldkotter M, Schwarzer V, Wirth R, Wienker TF, Wirth B. 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. Am J Hum Genet 2002;70:358-368.
Mailman MD, Heinz JW, Papp AC, Snyder PJ, Sedra MS, Wirth B, et al. Molecular analysis of spinal muscular atrophy and modification of the phenotype by SMN2. Genet Med 2002;4:20-26.
Prior TW, Swoboda KJ, Scott HD, Hejmanowski AQ. Homozygous SMN1 deletions in unaffected family members and modification of the phenotype by SMN2. Am J Med Genet A 2004;130:307-310.
Elsheikh B, Prior T, Zhang X, et al. An analysis of disease severity based on SMN2 copy number in adults with spinal muscular atrophy. Muscle Nerve 2009;40:652-656.
Leng Y, Chuang DM. Endogenous α-synuclein is induced by valproic acid through histone deacetylase inhibition and participates in neuroprotection against glutamate-induced excitotoxicity. J Neurosci 2006;26:7502-7512.
Burghes AH, McGovern VL. Antisense oligonucleotides and spinal muscular atrophy: skipping along. Genes Dev 2010;24:1574-1579.
Swoboda KJ, Scott CB, Crawford TO, Simard LR, Reyna SP, Krosschell KJ, et al. SMA CARNI-VAL Trial Part 1: double-blind, randomized, placebo-controlled trial of L-carnitine and valproic acid in spinal muscular atrophy. PLoS ONE 2010;5:e12140.
Burghes AHM, Beatty CE. Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick? Nat Rev Neurosci 2009;10:597-609.
Sproule DM, Montes J, Montgomery M, Battista V, Koenigsberger D, Shen W, et al. Increased fat mass and high incidence of overweight despite low body mass index in patients with spinal muscular atrophy. Neuromuscul Disord 2009;19:391-396.
Kolb S, Kissel JT. Spinal muscular atrophy: a timely review. Arch Neurol 2011;68:978-984.
Lefebvre S, Burlet P, Liu Q, Bertrandy S, Clermont O, Munnich A, et al. Correlation between severity and SMN protein level in spinal muscular atrophy. Nat Genet 1997;16:265-269.
van Bergeijk J, Haastert K, Grothe C, Claus P. Valproic acid promotes neurite outgrowth in PC12 cells independent from regulation of the survival of motoneuron protein. Chem Biol Drug Des 2006;67:244-247.
Darbar IA, Plaggert PG, Zanoteli E, Resende MBD, Reed UC. Evaluation of muscle strength and motor abilities in children with type II and III spinal muscular atrophy treated with valproic acid. BMC Neurol 2011;11:36.
Crawford TO, Paushkin SV, Kobayashi DT, Forrest SJ, Joyce CL, Finkel RS, et al. Evaluation of SMN protein, transcript and copy number in the biomarkers for spinal muscular atrophy (BforSMA) clinical study. PLoS ONE 2012;7:e33572.
Kernochan LE, Russo ML, Woodling NS, Huynh TN, Avila AM, Fischbeck KH, et al. The role of histone acetylation in SMN gene expression. Hum Mol Genet 2005;14:1171-1182.
Sproule DM, Montes, Dunaway S, Montgomery M, Battista V, Koenigsberger D, et al. Adiposity is increased among high-functioning, non-ambulatory patients with spinal muscular atrophy. Neuromuscul Disord 2010;20:448-452.
Finkel RS, Crawford TO, Swoboda KJ, Kaufmann P, Juhasz P, Li X, et al. Candidate proteins, metabolites, and transcripts in the biomarkers for spinal muscular atrophy (BforSMA) clinical study. PLoS ONE 2012;7:e35462.
MacKenzie A. Sense in antisense therapy for spinal muscular atrophy. N Engl J Med 2012;366:761-763.
Prior TW, Krainer AR, Hua Y, Swoboda KJ, Snyder PC, Bridgeman SJ, et al. A positive modifier of spinal muscular atrophy in the SMN2 gene. Am J Hum Genet 2009;85:408-413.
Sumner CJ, Huynh TN, Markowitz JA, et al. Valproic acid increases SMN levels in spinal muscular atrophy patient cells. Ann Neurol 2003;54:647-654.
Weihl CC, Connolly AM, Pestronk A. Valproate may improve strength and function in patients with type III/IV spinal muscular atrophy. Neurology 2006;67:500-501.
Swoboda KJ, Prior TW, Scott CB, McNaught TP, Wride MC, Reyna SB, et al. Natural history of denervation in SMA: relation to age, SMN2 copy number, and function. Ann Neurol 2005;57:704-712.
Tsai LK, Tsai MS, Ting CH, Li H. Multiple therapeutic effects of valproic acid in spinal muscular atrophy model mice. J Mol Med 2008;86:1243-1254.
McGuire D, Garrison L, Miller RG. Relationship of the Tufts Quantitative Neuromuscular Exam (TQNE) and the Sickness Impact Profile (SIP) in measuring progression of ALS. Neurology 1996;46:1442-1444.
Bebee TW, Doninguez CE, Chandler DS. Mouse models of SMA: tools for disease characterization and therapeutic development. Hum Genet 2012;131:1277-1293.
Markowitz JA, Singh P, Darras BT. Spinal muscular atrophy: a clinical and research update. Pediatr Neurol 2012:46:1-12.
Wirth B, Brichta L, Schrank B, Lochmüller H, Blick S, Baasner A, et al. Mildly affected patients with spinal muscular atrophy are partially protected by an increased SMN2 copy number. Hum Genet 2006;119:422-428.
Mercuri E, Bertini E, Iannaccone ST. Childhood spinal muscular atrophy: controversies and challenges. Lancet Neurol 2012:11:443-452.
Swoboda KJ, Scott CB, Reyna SP, Prior TW, LaSalle B, Sorenson SL, et al. Phase II open label study of valproic acid in spinal muscular atrophy. PLoS ONE 2009;4:e5268.
Brichta L, Hofmann Y, Hahnen E, Siebzehnrubl FA, Raschke H, Blumcke I, et al. Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy. Hum Mol Genet 2003;12:2481-2489.
Brichta L, Holker I, Haug K, Klockgether T, Wirth B. In vivo activation of SMN in spinal muscular atrophy carriers and patients treated with valproate. Ann Neurol 2006;59:970-975.
Kissel JT, Scott CB, Reyna SP, et al. SMA CARNI-VAL Trial Part II: a prospective, single-armed trial of L-carnitine and valproic acid in ambulatory children with spinal muscular atrophy. PLoS One 2011;6:e21296.
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References_xml – reference: Sproule DM, Montes, Dunaway S, Montgomery M, Battista V, Koenigsberger D, et al. Adiposity is increased among high-functioning, non-ambulatory patients with spinal muscular atrophy. Neuromuscul Disord 2010;20:448-452.
– reference: Weihl CC, Connolly AM, Pestronk A. Valproate may improve strength and function in patients with type III/IV spinal muscular atrophy. Neurology 2006;67:500-501.
– reference: Swoboda KJ, Scott CB, Reyna SP, Prior TW, LaSalle B, Sorenson SL, et al. Phase II open label study of valproic acid in spinal muscular atrophy. PLoS ONE 2009;4:e5268.
– reference: Sumner CJ, Huynh TN, Markowitz JA, et al. Valproic acid increases SMN levels in spinal muscular atrophy patient cells. Ann Neurol 2003;54:647-654.
– reference: Crawford TO, Paushkin SV, Kobayashi DT, Forrest SJ, Joyce CL, Finkel RS, et al. Evaluation of SMN protein, transcript and copy number in the biomarkers for spinal muscular atrophy (BforSMA) clinical study. PLoS ONE 2012;7:e33572.
– reference: Lorson CL, Rindt H, Shababi M. Spinal muscular atrophy: mechanisms and therapeutic strategies. Hum Mol Genet 2010;19:R111-R118.
– reference: Kolb S, Kissel JT. Spinal muscular atrophy: a timely review. Arch Neurol 2011;68:978-984.
– reference: Brichta L, Hofmann Y, Hahnen E, Siebzehnrubl FA, Raschke H, Blumcke I, et al. Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy. Hum Mol Genet 2003;12:2481-2489.
– reference: Burghes AH, McGovern VL. Antisense oligonucleotides and spinal muscular atrophy: skipping along. Genes Dev 2010;24:1574-1579.
– reference: Kernochan LE, Russo ML, Woodling NS, Huynh TN, Avila AM, Fischbeck KH, et al. The role of histone acetylation in SMN gene expression. Hum Mol Genet 2005;14:1171-1182.
– reference: Kissel JT, Scott CB, Reyna SP, et al. SMA CARNI-VAL Trial Part II: a prospective, single-armed trial of L-carnitine and valproic acid in ambulatory children with spinal muscular atrophy. PLoS One 2011;6:e21296.
– reference: McGuire D, Garrison L, Miller RG. Relationship of the Tufts Quantitative Neuromuscular Exam (TQNE) and the Sickness Impact Profile (SIP) in measuring progression of ALS. Neurology 1996;46:1442-1444.
– reference: Burghes AHM, Beatty CE. Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick? Nat Rev Neurosci 2009;10:597-609.
– reference: Mercuri E, Bertini E, Iannaccone ST. Childhood spinal muscular atrophy: controversies and challenges. Lancet Neurol 2012:11:443-452.
– reference: Sproule DM, Montes J, Montgomery M, Battista V, Koenigsberger D, Shen W, et al. Increased fat mass and high incidence of overweight despite low body mass index in patients with spinal muscular atrophy. Neuromuscul Disord 2009;19:391-396.
– reference: Wirth B, Brichta L, Schrank B, Lochmüller H, Blick S, Baasner A, et al. Mildly affected patients with spinal muscular atrophy are partially protected by an increased SMN2 copy number. Hum Genet 2006;119:422-428.
– reference: Leng Y, Chuang DM. Endogenous α-synuclein is induced by valproic acid through histone deacetylase inhibition and participates in neuroprotection against glutamate-induced excitotoxicity. J Neurosci 2006;26:7502-7512.
– reference: Brichta L, Holker I, Haug K, Klockgether T, Wirth B. In vivo activation of SMN in spinal muscular atrophy carriers and patients treated with valproate. Ann Neurol 2006;59:970-975.
– reference: Mailman MD, Heinz JW, Papp AC, Snyder PJ, Sedra MS, Wirth B, et al. Molecular analysis of spinal muscular atrophy and modification of the phenotype by SMN2. Genet Med 2002;4:20-26.
– reference: Darbar IA, Plaggert PG, Zanoteli E, Resende MBD, Reed UC. Evaluation of muscle strength and motor abilities in children with type II and III spinal muscular atrophy treated with valproic acid. BMC Neurol 2011;11:36.
– reference: Finkel RS, Crawford TO, Swoboda KJ, Kaufmann P, Juhasz P, Li X, et al. Candidate proteins, metabolites, and transcripts in the biomarkers for spinal muscular atrophy (BforSMA) clinical study. PLoS ONE 2012;7:e35462.
– reference: Markowitz JA, Singh P, Darras BT. Spinal muscular atrophy: a clinical and research update. Pediatr Neurol 2012:46:1-12.
– reference: Feldkotter M, Schwarzer V, Wirth R, Wienker TF, Wirth B. 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. Am J Hum Genet 2002;70:358-368.
– reference: Tsai LK, Tsai MS, Ting CH, Li H. Multiple therapeutic effects of valproic acid in spinal muscular atrophy model mice. J Mol Med 2008;86:1243-1254.
– reference: Bebee TW, Doninguez CE, Chandler DS. Mouse models of SMA: tools for disease characterization and therapeutic development. Hum Genet 2012;131:1277-1293.
– reference: Lefebvre S, Burlet P, Liu Q, Bertrandy S, Clermont O, Munnich A, et al. Correlation between severity and SMN protein level in spinal muscular atrophy. Nat Genet 1997;16:265-269.
– reference: Tsai LK, Yang CC, Hwu WL, Li H. Valproic acid treatment in six patients with spinal muscular atrophy. Eur J Neurol 2007;14:e8-e9.
– reference: Swoboda KJ, Prior TW, Scott CB, McNaught TP, Wride MC, Reyna SB, et al. Natural history of denervation in SMA: relation to age, SMN2 copy number, and function. Ann Neurol 2005;57:704-712.
– reference: Prior TW, Krainer AR, Hua Y, Swoboda KJ, Snyder PC, Bridgeman SJ, et al. A positive modifier of spinal muscular atrophy in the SMN2 gene. Am J Hum Genet 2009;85:408-413.
– reference: Elsheikh B, Prior T, Zhang X, et al. An analysis of disease severity based on SMN2 copy number in adults with spinal muscular atrophy. Muscle Nerve 2009;40:652-656.
– reference: Prior TW, Swoboda KJ, Scott HD, Hejmanowski AQ. Homozygous SMN1 deletions in unaffected family members and modification of the phenotype by SMN2. Am J Med Genet A 2004;130:307-310.
– reference: Swoboda KJ, Scott CB, Crawford TO, Simard LR, Reyna SP, Krosschell KJ, et al. SMA CARNI-VAL Trial Part 1: double-blind, randomized, placebo-controlled trial of L-carnitine and valproic acid in spinal muscular atrophy. PLoS ONE 2010;5:e12140.
– reference: van Bergeijk J, Haastert K, Grothe C, Claus P. Valproic acid promotes neurite outgrowth in PC12 cells independent from regulation of the survival of motoneuron protein. Chem Biol Drug Des 2006;67:244-247.
– reference: MacKenzie A. Sense in antisense therapy for spinal muscular atrophy. N Engl J Med 2012;366:761-763.
– volume: 85
  start-page: 408
  year: 2009
  end-page: 413
  article-title: A positive modifier of spinal muscular atrophy in the SMN2 gene
  publication-title: Am J Hum Genet
– volume: 366
  start-page: 761
  year: 2012
  end-page: 763
  article-title: Sense in antisense therapy for spinal muscular atrophy
  publication-title: N Engl J Med
– volume: 119
  start-page: 422
  year: 2006
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  article-title: Mildly affected patients with spinal muscular atrophy are partially protected by an increased SMN2 copy number
  publication-title: Hum Genet
– volume: 131
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  year: 2012
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  article-title: Mouse models of SMA: tools for disease characterization and therapeutic development
  publication-title: Hum Genet
– volume: 68
  start-page: 978
  year: 2011
  end-page: 984
  article-title: Spinal muscular atrophy: a timely review
  publication-title: Arch Neurol
– volume: 5
  start-page: e12140
  year: 2010
  article-title: SMA CARNI‐VAL Trial Part 1: double‐blind, randomized, placebo‐controlled trial of L‐carnitine and valproic acid in spinal muscular atrophy
  publication-title: PLoS ONE
– volume: 57
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  article-title: Natural history of denervation in SMA: relation to age, SMN2 copy number, and function
  publication-title: Ann Neurol
– volume: 46
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  year: 1996
  end-page: 1444
  article-title: Relationship of the Tufts Quantitative Neuromuscular Exam (TQNE) and the Sickness Impact Profile (SIP) in measuring progression of ALS
  publication-title: Neurology
– volume: 11
  start-page: 443
  year: 2012
  end-page: 452
  article-title: Childhood spinal muscular atrophy: controversies and challenges
  publication-title: Lancet Neurol
– volume: 14
  start-page: e8
  year: 2007
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  article-title: Valproic acid treatment in six patients with spinal muscular atrophy
  publication-title: Eur J Neurol
– volume: 67
  start-page: 244
  year: 2006
  end-page: 247
  article-title: Valproic acid promotes neurite outgrowth in PC12 cells independent from regulation of the survival of motoneuron protein
  publication-title: Chem Biol Drug Des
– volume: 4
  start-page: 20
  year: 2002
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  article-title: Molecular analysis of spinal muscular atrophy and modification of the phenotype by SMN2
  publication-title: Genet Med
– volume: 67
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  year: 2006
  end-page: 501
  article-title: Valproate may improve strength and function in patients with type III/IV spinal muscular atrophy
  publication-title: Neurology
– volume: 6
  start-page: e21296
  year: 2011
  article-title: SMA CARNI‐VAL Trial Part II: a prospective, single‐armed trial of L‐carnitine and valproic acid in ambulatory children with spinal muscular atrophy
  publication-title: PLoS One
– volume: 10
  start-page: 597
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Snippet ABSTRACT Introduction: An open‐label trial suggested that valproic acid (VPA) improved strength in adults with spinal muscular atrophy (SMA). We report a...
Introduction : An open‐label trial suggested that valproic acid (VPA) improved strength in adults with spinal muscular atrophy (SMA). We report a 12‐month,...
An open-label trial suggested that valproic acid (VPA) improved strength in adults with spinal muscular atrophy (SMA). We report a 12-month, double-blind,...
Introduction: An open-label trial suggested that valproic acid (VPA) improved strength in adults with spinal muscular atrophy (SMA). We report a 12-month,...
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StartPage 187
SubjectTerms Adult
Ambulatory Care
carnitine
Cohort Studies
Cross-Over Studies
Dose-Response Relationship, Drug
Double-Blind Method
Female
Histone Deacetylase Inhibitors - pharmacology
Histone Deacetylase Inhibitors - therapeutic use
Humans
Male
Middle Aged
motor neuron disease
Muscle Contraction - drug effects
Muscle Contraction - physiology
Muscle Strength - drug effects
Muscle Strength - physiology
Muscular Atrophy, Spinal - drug therapy
Muscular Atrophy, Spinal - physiopathology
Muscular system
Prospective Studies
spinal muscular atrophy
Treatment Outcome
valproic acid
Valproic Acid - pharmacology
Valproic Acid - therapeutic use
Title SMA valiant trial: A prospective, double-blind, placebo-controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy
URI https://api.istex.fr/ark:/67375/WNG-XD4CJMBG-2/fulltext.pdf
https://onlinelibrary.wiley.com/doi/abs/10.1002%2Fmus.23904
https://www.ncbi.nlm.nih.gov/pubmed/23681940
https://www.proquest.com/docview/1477699627
https://www.proquest.com/docview/1503547956
https://www.proquest.com/docview/1534824894
https://pubmed.ncbi.nlm.nih.gov/PMC3888833
Volume 49
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