valproic acid has been researched along with Muscular Atrophy, Spinal in 28 studies
Valproic Acid: A fatty acid with anticonvulsant and anti-manic properties that is used in the treatment of EPILEPSY and BIPOLAR DISORDER. The mechanisms of its therapeutic actions are not well understood. It may act by increasing GAMMA-AMINOBUTYRIC ACID levels in the brain or by altering the properties of VOLTAGE-GATED SODIUM CHANNELS.
valproic acid : A branched-chain saturated fatty acid that comprises of a propyl substituent on a pentanoic acid stem.
Muscular Atrophy, Spinal: A group of disorders marked by progressive degeneration of motor neurons in the spinal cord resulting in weakness and muscular atrophy, usually without evidence of injury to the corticospinal tracts. Diseases in this category include Werdnig-Hoffmann disease and later onset SPINAL MUSCULAR ATROPHIES OF CHILDHOOD, most of which are hereditary. (Adams et al., Principles of Neurology, 6th ed, p1089)
| Excerpt | Relevance | Reference |
|---|---|---|
| " The validity of this approach remains uncertain and could be improved by understanding sources of pharmacokinetic variability." | 6.77 | Population pharmacokinetics of valproic acid in pediatric patients with epilepsy: considerations for dosing spinal muscular atrophy patients. ( Barrett, JS; Jayaraman, B; Swoboda, KJ; Williams, JH, 2012) |
| " The validity of this approach remains uncertain and could be improved by understanding sources of pharmacokinetic variability." | 2.77 | Population pharmacokinetics of valproic acid in pediatric patients with epilepsy: considerations for dosing spinal muscular atrophy patients. ( Barrett, JS; Jayaraman, B; Swoboda, KJ; Williams, JH, 2012) |
| "Valproic acid (VPA) has demonstrated potential as a therapeutic candidate for spinal muscular atrophy (SMA) in vitro and in vivo." | 2.75 | SMA CARNI-VAL trial part I: double-blind, randomized, placebo-controlled trial of L-carnitine and valproic acid in spinal muscular atrophy. ( Acsadi, G; Bromberg, MB; Chan, GM; Crawford, TO; D'Anjou, G; Elsheik, B; Kissel, JT; Krosschell, KJ; LaSalle, B; Maczulski, JA; Prior, TW; Reyna, SP; Schroth, MK; Scott, CB; Simard, LR; Sorenson, SL; Swoboda, KJ, 2010) |
| "Blood was collected from 10 spinal muscular atrophy carriers and 20 spinal muscular atrophy patients treated with valproic acid." | 2.72 | In vivo activation of SMN in spinal muscular atrophy carriers and patients treated with valproate. ( Brichta, L; Haug, K; Holker, I; Klockgether, T; Wirth, B, 2006) |
| "Valproic acid (VPA) is a histone deacetylase (HDAC) inhibitor that has shown positive results on SMA both in experimental and cohort studies." | 2.61 | Efficacy and Safety of Valproic Acid for Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis. ( Abo-Elghar, H; Doheim, MF; ELdoadoa, MF; Elshafay, A; Hieu, TH; Hirayama, K; Holloway, SK; Huy, NT; Kassem, MAM, 2019) |
| "Spinal muscular atrophy is a devastating disease that is characterized by degeneration and death of a specific subclass of motor neurons in the anterior horn of the spinal cord." | 1.43 | Neuron-specific knock-down of SMN1 causes neuron degeneration and death through an apoptotic mechanism. ( Battaglia, GS; Bazzicalupo, P; Castro, S; Chaplin, JC; Di Schiavi, E; Donato, A; Esposito, A; Gallotta, I; Hilliard, MA; Mazzarella, N; Zampi, G, 2016) |
| "Valproic acid was prescribed in 20mg/kg/day during 2 weeks." | 1.40 | [Effect of valproic acid on SMN protein level in peripheral blood mononuclear cells of patients with spinal muscular atrophy and different SMN2 copy numbers]. ( Koliada, AK; Shatilo, AV; Sokolik, VV, 2014) |
| "Functional loss of SMN1 causes proximal spinal muscular atrophy (SMA), the most common genetic condition accounting for infant lethality." | 1.39 | VPA response in SMA is suppressed by the fatty acid translocase CD36. ( Bauer, T; Brüstle, O; Dimos, J; Garbes, L; Heesen, L; Heller, R; Hölker, I; Peitz, M; Schreml, J; Thoenes, M; Walter, M; Wirth, B; Zimmermann, K, 2013) |
| "Valproic acid (VPA) is a histone deacetylase inhibitor that can increase SMN levels in some SMA cells or SMA patients through activation of SMN2 transcription or splicing correction of SMN2 exon 7." | 1.38 | Valproic acid increases SMN2 expression and modulates SF2/ASF and hnRNPA1 expression in SMA fibroblast cell lines. ( Harahap, IS; Lee, MJ; Matsuo, M; Morikawa, S; Nishimura, N; Nishio, H; Nurputra, DK; Saito, T; San, LP; Sasaki, N; Takeshima, Y; Yamamoto, T; Yusoff, S, 2012) |
| "Proximal spinal muscular atrophy (SMA) is a common neuromuscular disorder causing infant death in half of all patients." | 1.32 | Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy. ( Blumcke, I; Brichta, L; Eyupoglu, IY; Hahnen, E; Hofmann, Y; Raschke, H; Siebzehnrubl, FA; Wirth, B, 2003) |
| Timeframe | Studies, this research(%) | All Research% |
|---|---|---|
| pre-1990 | 0 (0.00) | 18.7374 |
| 1990's | 0 (0.00) | 18.2507 |
| 2000's | 14 (50.00) | 29.6817 |
| 2010's | 14 (50.00) | 24.3611 |
| 2020's | 0 (0.00) | 2.80 |
| Authors | Studies |
|---|---|
| Farrelly-Rosch, A | 1 |
| Lau, CL | 1 |
| Patil, N | 1 |
| Turner, BJ | 1 |
| Shabanpoor, F | 1 |
| Elshafay, A | 1 |
| Hieu, TH | 1 |
| Doheim, MF | 1 |
| Kassem, MAM | 1 |
| ELdoadoa, MF | 1 |
| Holloway, SK | 1 |
| Abo-Elghar, H | 1 |
| Hirayama, K | 1 |
| Huy, NT | 1 |
| Kissel, JT | 4 |
| Elsheikh, B | 2 |
| King, WM | 1 |
| Freimer, M | 1 |
| Scott, CB | 4 |
| Kolb, SJ | 1 |
| Reyna, SP | 4 |
| Crawford, TO | 4 |
| Simard, LR | 4 |
| Krosschell, KJ | 4 |
| Acsadi, G | 4 |
| Schroth, MK | 4 |
| D'Anjou, G | 4 |
| LaSalle, B | 4 |
| Prior, TW | 4 |
| Sorenson, S | 2 |
| Maczulski, JA | 3 |
| Swoboda, KJ | 5 |
| Singh, P | 1 |
| Liew, WK | 1 |
| Darras, BT | 1 |
| Sokolik, VV | 1 |
| Koliada, AK | 1 |
| Shatilo, AV | 1 |
| Yoshida, M | 1 |
| Kitaoka, S | 1 |
| Egawa, N | 1 |
| Yamane, M | 1 |
| Ikeda, R | 1 |
| Tsukita, K | 1 |
| Amano, N | 1 |
| Watanabe, A | 1 |
| Morimoto, M | 1 |
| Takahashi, J | 1 |
| Hosoi, H | 1 |
| Nakahata, T | 1 |
| Inoue, H | 1 |
| Saito, MK | 1 |
| Gallotta, I | 1 |
| Mazzarella, N | 1 |
| Donato, A | 1 |
| Esposito, A | 1 |
| Chaplin, JC | 1 |
| Castro, S | 1 |
| Zampi, G | 1 |
| Battaglia, GS | 1 |
| Hilliard, MA | 1 |
| Bazzicalupo, P | 1 |
| Di Schiavi, E | 1 |
| Humphrey, E | 1 |
| Lam, LT | 1 |
| Fuller, HR | 2 |
| Lynch, TA | 1 |
| Sewry, CA | 1 |
| Goodwin, PR | 1 |
| Mackenzie, AE | 1 |
| Morris, GE | 2 |
| Tsai, LK | 3 |
| Tsai, MS | 2 |
| Ting, CH | 1 |
| Li, H | 3 |
| Mattis, VB | 1 |
| Butchbach, ME | 1 |
| Lorson, CL | 1 |
| Bönnemann, CG | 1 |
| Finkel, RS | 1 |
| Baranov, VS | 1 |
| Kiselev, AV | 1 |
| Vakharlovskiĭ, VG | 1 |
| Zhelezniakova, GIu | 1 |
| Komantsev, VN | 1 |
| Malysheva, OV | 1 |
| Glotov, AS | 1 |
| Ivashchenko, TE | 1 |
| Baranov, AN | 1 |
| Sorenson, SL | 2 |
| Wood, J | 1 |
| Bromberg, MB | 3 |
| Chan, GM | 3 |
| Garbes, L | 2 |
| Riessland, M | 1 |
| Hölker, I | 3 |
| Heller, R | 2 |
| Hauke, J | 1 |
| Tränkle, C | 1 |
| Coras, R | 1 |
| Blümcke, I | 2 |
| Hahnen, E | 2 |
| Wirth, B | 4 |
| Man, NT | 1 |
| Lam, le T | 1 |
| Shamanin, VA | 1 |
| Androphy, EJ | 1 |
| Elsheik, B | 2 |
| Harahap, IS | 1 |
| Saito, T | 1 |
| San, LP | 1 |
| Sasaki, N | 1 |
| Nurputra, DK | 1 |
| Yusoff, S | 1 |
| Yamamoto, T | 1 |
| Morikawa, S | 1 |
| Nishimura, N | 1 |
| Lee, MJ | 1 |
| Takeshima, Y | 1 |
| Matsuo, M | 1 |
| Nishio, H | 1 |
| Also-Rallo, E | 1 |
| Alías, L | 1 |
| Martínez-Hernández, R | 1 |
| Caselles, L | 1 |
| Barceló, MJ | 1 |
| Baiget, M | 1 |
| Bernal, S | 1 |
| Tizzano, EF | 1 |
| Williams, JH | 1 |
| Jayaraman, B | 1 |
| Barrett, JS | 1 |
| Heesen, L | 1 |
| Bauer, T | 1 |
| Schreml, J | 1 |
| Zimmermann, K | 1 |
| Thoenes, M | 1 |
| Walter, M | 1 |
| Dimos, J | 1 |
| Peitz, M | 1 |
| Brüstle, O | 1 |
| Brichta, L | 2 |
| Hofmann, Y | 1 |
| Siebzehnrubl, FA | 1 |
| Raschke, H | 1 |
| Eyupoglu, IY | 1 |
| Kernochan, LE | 1 |
| Russo, ML | 1 |
| Woodling, NS | 1 |
| Huynh, TN | 1 |
| Avila, AM | 1 |
| Fischbeck, KH | 1 |
| Sumner, CJ | 1 |
| Haug, K | 1 |
| Klockgether, T | 1 |
| van Bergeijk, J | 1 |
| Haastert, K | 1 |
| Grothe, C | 1 |
| Claus, P | 1 |
| Weihl, CC | 1 |
| Connolly, AM | 1 |
| Pestronk, A | 1 |
| Lin, TB | 1 |
| Hwu, WL | 2 |
| Yang, CC | 1 |
| Trial | Phase | Enrollment | Study Type | Start Date | Status | ||
|---|---|---|---|---|---|---|---|
| In Vivo Study of Safety, Tolerability and Dosing Effect on SMN mRNA and Protein Levels of Valproic Acid in Patients With Spinal Muscular Atrophy[NCT00374075] | Phase 1 | 42 participants | Interventional | 2003-09-30 | Completed | ||
| Prospective Evaluation of Infants With Spinal Muscular Atrophy: SPOT SMA[NCT02831296] | 1,000 participants (Anticipated) | Observational [Patient Registry] | 2016-02-29 | Recruiting | |||
| Evaluation of the Muscle Strength and Motor Ability in Children With Spinal Muscle Atrophy Type II and III Treated With Valproic Acid[NCT01033331] | 22 participants (Actual) | Observational | 2006-07-31 | Completed | |||
| Multi-center Phase II Trial of Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy (SMA CARNI-VAL Trial)[NCT00227266] | Phase 2 | 94 participants (Actual) | Interventional | 2005-09-30 | Completed | ||
| Phase I/II Trial of Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy Type I (CARNI-VAL Type I)[NCT00661453] | Phase 1/Phase 2 | 40 participants (Actual) | Interventional | 2008-04-30 | Completed | ||
| [information is prepared from clinicaltrials.gov, extracted Sep-2024] | |||||||
The maximum Compound Motor Action Potential (CMAP) is a measurement obtained through EMG testing that is associated with disease progression. In this study, we measure the maximum CMAP by stimulating one nerve in the hand and measuring the response of the muscle. This is done multiple times, the outcome used is the highest peak, or response observed. (NCT00227266)
Timeframe: 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available)
| Intervention | mV (Mean) | |
|---|---|---|
| Baseline | 6 months | |
| Cohort 1a Sitters Placebo Then Treatment | 2.28 | 2.32 |
| Cohort 1b Sitters Treatment | 2.93 | 2.37 |
| Cohort 2 Standers and Walkers - Treatment | 5.52 | 6.56 |
The maximum Compound Motor Action Potential (CMAP) is a measurement obtained through EMG testing that is associated with disease progression. In this study, we measure the maximum CMAP by stimulating one nerve in the hand and measuring the response of the muscle. This is done multiple times, the outcome used is the highest peak, or response observed. (NCT00227266)
Timeframe: 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available)
| Intervention | mV (Median) | |
|---|---|---|
| Baseline | 6 months | |
| Cohort 1a Sitters Placebo Then Treatment | 1.91 | 1.44 |
| Cohort 1b Sitters Treatment | 2.2 | 1.8 |
| Cohort 2 Standers and Walkers - Treatment | 5.3 | 5.85 |
The maximum Compound Motor Action Potential (CMAP) area is a measurement obtained through EMG testing that is associated with disease progression. In this study, we measure the maximum CMAP by stimulating one nerve in the hand and measuring the response of the muscle. This procedure is repeated multiple times. The maximum area is the response that results in the largest area under the response curve. (NCT00227266)
Timeframe: 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available)
| Intervention | mVms (Mean) | |
|---|---|---|
| Baseline | 6 months | |
| Cohort 1a Sitters Placebo Then Treatment | 5.46 | 5.28 |
| Cohort 1b Sitters Treatment | 5.45 | 5.26 |
| Cohort 2 Standers and Walkers - Treatment | 14.85 | 16.26 |
The maximum Compound Motor Action Potential (CMAP) area is a measurement obtained through EMG testing that is associated with disease progression. In this study, we measure the maximum CMAP by stimulating one nerve in the hand and measuring the response of the muscle. This procedure is repeated multiple times. The maximum area is the response that results in the largest area under the response curve. (NCT00227266)
Timeframe: 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available)
| Intervention | mVms (Median) | |
|---|---|---|
| Baseline | 6 months | |
| Cohort 1a Sitters Placebo Then Treatment | 3.6 | 3.74 |
| Cohort 1b Sitters Treatment | 4.6 | 3.4 |
| Cohort 2 Standers and Walkers - Treatment | 13.65 | 16.85 |
Comparison of Modified Hammersmith Change from baseline to 6 months. Scores range from 0 to 40. A higher score indicates a better outcome. This scale is used to assess gross motor abilities of non-ambulant children with SMA in multiple research trials as well as in clinical settings. (NCT00227266)
Timeframe: 0 months, 6 months
| Intervention | Score (Mean) | ||
|---|---|---|---|
| Baseline visit (0 weeks) | 6 Month visit (V2) | Change from Baseline | |
| Cohort 1a Sitters Placebo Then Treatment | 20.0 | 20.6 | 0.6 |
| Cohort 1b Sitters Treatment | 16.6 | 16.8 | 0.2 |
"Baseline Modified Hammersmith Extend testing. The baseline test is the score they receive during their screening visits. This scale ranges from 0 to 56. A higher score indicates a better outcome.~This scale is used to assess gross motor abilities of children with SMA in multiple research trials as well as in clinical settings." (NCT00227266)
Timeframe: 1 month prior to enrollment, at enrollment (0 months)
| Intervention | Score (Mean) | |
|---|---|---|
| Modified Hammersmith Extend at S1 (-4 weeks) | Modified Hammersmith Extend at S2 (0 weeks) | |
| Cohort 2 Experimental | 47.0 | 48.3 |
(NCT00661453)
Timeframe: -2 weeks, time 0, 3 months, 6 months
| Intervention | g (Mean) | |||||
|---|---|---|---|---|---|---|
| Lean Mass Baseline | Lean Mass 3 months | Lean Mass 6 months | Fat Mass Baseline | Fat Mass 3 months | Fat Mass 6 months | |
| SMA Type 1 | 4317.15 | 4993.92 | 5133.83 | 3011.37 | 3618.25 | 4316.08 |
3 reviews available for valproic acid and Muscular Atrophy, Spinal
| Article | Year |
|---|---|
Efficacy and Safety of Valproic Acid for Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis.
Topics: Gene Expression; Humans; Motor Activity; Motor Neurons; Muscular Atrophy, Spinal; Respiration; Survi | 2019 |
Efficacy and Safety of Valproic Acid for Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis.
Topics: Gene Expression; Humans; Motor Activity; Motor Neurons; Muscular Atrophy, Spinal; Respiration; Survi | 2019 |
Current advances in drug development in spinal muscular atrophy.
Topics: Animals; Child; Child, Preschool; Disease Models, Animal; Drug Design; Female; Genetic Therapy; Huma | 2013 |
Current advances in drug development in spinal muscular atrophy.
Topics: Animals; Child; Child, Preschool; Disease Models, Animal; Drug Design; Female; Genetic Therapy; Huma | 2013 |
[Molecular genetic basis of proximal spinal muscular atrophy and experience in its pharmaceutical treatment].
Topics: GABA Agents; Humans; Muscular Atrophy, Spinal; Ribonucleoproteins, Small Nuclear; SMN Complex Protei | 2008 |
[Molecular genetic basis of proximal spinal muscular atrophy and experience in its pharmaceutical treatment].
Topics: GABA Agents; Humans; Muscular Atrophy, Spinal; Ribonucleoproteins, Small Nuclear; SMN Complex Protei | 2008 |
6 trials available for valproic acid and Muscular Atrophy, Spinal
| Article | Year |
|---|---|
SMA valiant trial: a prospective, double-blind, placebo-controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy.
Topics: Adult; Ambulatory Care; Cohort Studies; Cross-Over Studies; Dose-Response Relationship, Drug; Double | 2014 |
SMA valiant trial: a prospective, double-blind, placebo-controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy.
Topics: Adult; Ambulatory Care; Cohort Studies; Cross-Over Studies; Dose-Response Relationship, Drug; Double | 2014 |
Phase II open label study of valproic acid in spinal muscular atrophy.
Topics: Absorptiometry, Photon; Adolescent; Adult; Analysis of Variance; Body Composition; Bone Density; Chi | 2009 |
Phase II open label study of valproic acid in spinal muscular atrophy.
Topics: Absorptiometry, Photon; Adolescent; Adult; Analysis of Variance; Body Composition; Bone Density; Chi | 2009 |
Phase II open label study of valproic acid in spinal muscular atrophy.
Topics: Absorptiometry, Photon; Adolescent; Adult; Analysis of Variance; Body Composition; Bone Density; Chi | 2009 |
Phase II open label study of valproic acid in spinal muscular atrophy.
Topics: Absorptiometry, Photon; Adolescent; Adult; Analysis of Variance; Body Composition; Bone Density; Chi | 2009 |
Phase II open label study of valproic acid in spinal muscular atrophy.
Topics: Absorptiometry, Photon; Adolescent; Adult; Analysis of Variance; Body Composition; Bone Density; Chi | 2009 |
Phase II open label study of valproic acid in spinal muscular atrophy.
Topics: Absorptiometry, Photon; Adolescent; Adult; Analysis of Variance; Body Composition; Bone Density; Chi | 2009 |
Phase II open label study of valproic acid in spinal muscular atrophy.
Topics: Absorptiometry, Photon; Adolescent; Adult; Analysis of Variance; Body Composition; Bone Density; Chi | 2009 |
Phase II open label study of valproic acid in spinal muscular atrophy.
Topics: Absorptiometry, Photon; Adolescent; Adult; Analysis of Variance; Body Composition; Bone Density; Chi | 2009 |
Phase II open label study of valproic acid in spinal muscular atrophy.
Topics: Absorptiometry, Photon; Adolescent; Adult; Analysis of Variance; Body Composition; Bone Density; Chi | 2009 |
Phase II open label study of valproic acid in spinal muscular atrophy.
Topics: Absorptiometry, Photon; Adolescent; Adult; Analysis of Variance; Body Composition; Bone Density; Chi | 2009 |
Phase II open label study of valproic acid in spinal muscular atrophy.
Topics: Absorptiometry, Photon; Adolescent; Adult; Analysis of Variance; Body Composition; Bone Density; Chi | 2009 |
Phase II open label study of valproic acid in spinal muscular atrophy.
Topics: Absorptiometry, Photon; Adolescent; Adult; Analysis of Variance; Body Composition; Bone Density; Chi | 2009 |
Phase II open label study of valproic acid in spinal muscular atrophy.
Topics: Absorptiometry, Photon; Adolescent; Adult; Analysis of Variance; Body Composition; Bone Density; Chi | 2009 |
Phase II open label study of valproic acid in spinal muscular atrophy.
Topics: Absorptiometry, Photon; Adolescent; Adult; Analysis of Variance; Body Composition; Bone Density; Chi | 2009 |
Phase II open label study of valproic acid in spinal muscular atrophy.
Topics: Absorptiometry, Photon; Adolescent; Adult; Analysis of Variance; Body Composition; Bone Density; Chi | 2009 |
Phase II open label study of valproic acid in spinal muscular atrophy.
Topics: Absorptiometry, Photon; Adolescent; Adult; Analysis of Variance; Body Composition; Bone Density; Chi | 2009 |
Phase II open label study of valproic acid in spinal muscular atrophy.
Topics: Absorptiometry, Photon; Adolescent; Adult; Analysis of Variance; Body Composition; Bone Density; Chi | 2009 |
Phase II open label study of valproic acid in spinal muscular atrophy.
Topics: Absorptiometry, Photon; Adolescent; Adult; Analysis of Variance; Body Composition; Bone Density; Chi | 2009 |
SMA CARNI-VAL trial part I: double-blind, randomized, placebo-controlled trial of L-carnitine and valproic acid in spinal muscular atrophy.
Topics: Age Factors; Body Composition; Body Mass Index; Body Weight; Bone Density; Carnitine; Child; Child, | 2010 |
SMA CARNI-VAL trial part I: double-blind, randomized, placebo-controlled trial of L-carnitine and valproic acid in spinal muscular atrophy.
Topics: Age Factors; Body Composition; Body Mass Index; Body Weight; Bone Density; Carnitine; Child; Child, | 2010 |
SMA CARNIVAL TRIAL PART II: a prospective, single-armed trial of L-carnitine and valproic acid in ambulatory children with spinal muscular atrophy.
Topics: Action Potentials; Adolescent; Anticonvulsants; Carnitine; Child; Child, Preschool; Demography; Fema | 2011 |
SMA CARNIVAL TRIAL PART II: a prospective, single-armed trial of L-carnitine and valproic acid in ambulatory children with spinal muscular atrophy.
Topics: Action Potentials; Adolescent; Anticonvulsants; Carnitine; Child; Child, Preschool; Demography; Fema | 2011 |
Population pharmacokinetics of valproic acid in pediatric patients with epilepsy: considerations for dosing spinal muscular atrophy patients.
Topics: Adolescent; Anticonvulsants; Capsules; Child; Child, Preschool; Epilepsy; Female; Humans; Infant; Ma | 2012 |
Population pharmacokinetics of valproic acid in pediatric patients with epilepsy: considerations for dosing spinal muscular atrophy patients.
Topics: Adolescent; Anticonvulsants; Capsules; Child; Child, Preschool; Epilepsy; Female; Humans; Infant; Ma | 2012 |
In vivo activation of SMN in spinal muscular atrophy carriers and patients treated with valproate.
Topics: Cyclic AMP Response Element-Binding Protein; GABA Agents; Gene Expression; Heterozygote; Humans; Mus | 2006 |
In vivo activation of SMN in spinal muscular atrophy carriers and patients treated with valproate.
Topics: Cyclic AMP Response Element-Binding Protein; GABA Agents; Gene Expression; Heterozygote; Humans; Mus | 2006 |
19 other studies available for valproic acid and Muscular Atrophy, Spinal
| Article | Year |
|---|---|
Combination of valproic acid and morpholino splice-switching oligonucleotide produces improved outcomes in spinal muscular atrophy patient-derived fibroblasts.
Topics: Cell Survival; Cells, Cultured; Dose-Response Relationship, Drug; Drug Therapy, Combination; Fibrobl | 2017 |
Combination of valproic acid and morpholino splice-switching oligonucleotide produces improved outcomes in spinal muscular atrophy patient-derived fibroblasts.
Topics: Cell Survival; Cells, Cultured; Dose-Response Relationship, Drug; Drug Therapy, Combination; Fibrobl | 2017 |
[Effect of valproic acid on SMN protein level in peripheral blood mononuclear cells of patients with spinal muscular atrophy and different SMN2 copy numbers].
Topics: Adult; beta 2-Microglobulin; Biomarkers; Child; Child, Preschool; Female; Gene Dosage; Genetic Marke | 2014 |
[Effect of valproic acid on SMN protein level in peripheral blood mononuclear cells of patients with spinal muscular atrophy and different SMN2 copy numbers].
Topics: Adult; beta 2-Microglobulin; Biomarkers; Child; Child, Preschool; Female; Gene Dosage; Genetic Marke | 2014 |
Modeling the early phenotype at the neuromuscular junction of spinal muscular atrophy using patient-derived iPSCs.
Topics: Animals; Cell Culture Techniques; Cell Differentiation; Cell Line; Coculture Techniques; Humans; Ind | 2015 |
Modeling the early phenotype at the neuromuscular junction of spinal muscular atrophy using patient-derived iPSCs.
Topics: Animals; Cell Culture Techniques; Cell Differentiation; Cell Line; Coculture Techniques; Humans; Ind | 2015 |
Neuron-specific knock-down of SMN1 causes neuron degeneration and death through an apoptotic mechanism.
Topics: Animals; Animals, Genetically Modified; Caenorhabditis elegans; Disease Models, Animal; Gene Knockdo | 2016 |
Neuron-specific knock-down of SMN1 causes neuron degeneration and death through an apoptotic mechanism.
Topics: Animals; Animals, Genetically Modified; Caenorhabditis elegans; Disease Models, Animal; Gene Knockdo | 2016 |
A two-site ELISA can quantify upregulation of SMN protein by drugs for spinal muscular atrophy.
Topics: Blotting, Western; Cell Line; Cell Survival; Central Nervous System Agents; Enzyme-Linked Immunosorb | 2008 |
A two-site ELISA can quantify upregulation of SMN protein by drugs for spinal muscular atrophy.
Topics: Blotting, Western; Cell Line; Cell Survival; Central Nervous System Agents; Enzyme-Linked Immunosorb | 2008 |
Multiple therapeutic effects of valproic acid in spinal muscular atrophy model mice.
Topics: Animals; Astrocytes; Cell Proliferation; Disease Models, Animal; Humans; Mice; Mice, Transgenic; Mot | 2008 |
Multiple therapeutic effects of valproic acid in spinal muscular atrophy model mice.
Topics: Animals; Astrocytes; Cell Proliferation; Disease Models, Animal; Humans; Mice; Mice, Transgenic; Mot | 2008 |
Detection of human survival motor neuron (SMN) protein in mice containing the SMN2 transgene: applicability to preclinical therapy development for spinal muscular atrophy.
Topics: Alternative Splicing; Amino Acid Sequence; Animals; Antibodies, Monoclonal; Cells, Cultured; Disease | 2008 |
Detection of human survival motor neuron (SMN) protein in mice containing the SMN2 transgene: applicability to preclinical therapy development for spinal muscular atrophy.
Topics: Alternative Splicing; Amino Acid Sequence; Animals; Antibodies, Monoclonal; Cells, Cultured; Disease | 2008 |
Linking SMN to SMA: an assay for the rescuer.
Topics: Cell Survival; Central Nervous System Agents; Enzyme-Linked Immunosorbent Assay; Humans; Hydroxyurea | 2008 |
Linking SMN to SMA: an assay for the rescuer.
Topics: Cell Survival; Central Nervous System Agents; Enzyme-Linked Immunosorbent Assay; Humans; Hydroxyurea | 2008 |
LBH589 induces up to 10-fold SMN protein levels by several independent mechanisms and is effective even in cells from SMA patients non-responsive to valproate.
Topics: Animals; Cells, Cultured; Fibroblasts; Gene Expression; Humans; Hydroxamic Acids; Indoles; Mice; Mic | 2009 |
LBH589 induces up to 10-fold SMN protein levels by several independent mechanisms and is effective even in cells from SMA patients non-responsive to valproate.
Topics: Animals; Cells, Cultured; Fibroblasts; Gene Expression; Humans; Hydroxamic Acids; Indoles; Mice; Mic | 2009 |
Valproate and bone loss: iTRAQ proteomics show that valproate reduces collagens and osteonectin in SMA cells.
Topics: Bone Diseases, Metabolic; Chromatography, Liquid; Collagen; Electrophoresis, Polyacrylamide Gel; Fib | 2010 |
Valproate and bone loss: iTRAQ proteomics show that valproate reduces collagens and osteonectin in SMA cells.
Topics: Bone Diseases, Metabolic; Chromatography, Liquid; Collagen; Electrophoresis, Polyacrylamide Gel; Fib | 2010 |
Valproic acid increases SMN2 expression and modulates SF2/ASF and hnRNPA1 expression in SMA fibroblast cell lines.
Topics: Adult; Blotting, Western; Cell Line; Fibroblasts; Gene Expression; Heterogeneous Nuclear Ribonucleop | 2012 |
Valproic acid increases SMN2 expression and modulates SF2/ASF and hnRNPA1 expression in SMA fibroblast cell lines.
Topics: Adult; Blotting, Western; Cell Line; Fibroblasts; Gene Expression; Heterogeneous Nuclear Ribonucleop | 2012 |
Treatment of spinal muscular atrophy cells with drugs that upregulate SMN expression reveals inter- and intra-patient variability.
Topics: Cell Line, Transformed; Cells, Cultured; Drug Resistance; Female; Fibroblasts; Frameshift Mutation; | 2011 |
Treatment of spinal muscular atrophy cells with drugs that upregulate SMN expression reveals inter- and intra-patient variability.
Topics: Cell Line, Transformed; Cells, Cultured; Drug Resistance; Female; Fibroblasts; Frameshift Mutation; | 2011 |
VPA response in SMA is suppressed by the fatty acid translocase CD36.
Topics: CD36 Antigens; Cell Line; Fibroblasts; GABAergic Neurons; Gene Expression Profiling; Humans; Muscula | 2013 |
VPA response in SMA is suppressed by the fatty acid translocase CD36.
Topics: CD36 Antigens; Cell Line; Fibroblasts; GABAergic Neurons; Gene Expression Profiling; Humans; Muscula | 2013 |
Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy.
Topics: Animals; Antibodies, Monoclonal; Blotting, Western; Cells, Cultured; Dose-Response Relationship, Dru | 2003 |
Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy.
Topics: Animals; Antibodies, Monoclonal; Blotting, Western; Cells, Cultured; Dose-Response Relationship, Dru | 2003 |
Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy.
Topics: Animals; Antibodies, Monoclonal; Blotting, Western; Cells, Cultured; Dose-Response Relationship, Dru | 2003 |
Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy.
Topics: Animals; Antibodies, Monoclonal; Blotting, Western; Cells, Cultured; Dose-Response Relationship, Dru | 2003 |
Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy.
Topics: Animals; Antibodies, Monoclonal; Blotting, Western; Cells, Cultured; Dose-Response Relationship, Dru | 2003 |
Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy.
Topics: Animals; Antibodies, Monoclonal; Blotting, Western; Cells, Cultured; Dose-Response Relationship, Dru | 2003 |
Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy.
Topics: Animals; Antibodies, Monoclonal; Blotting, Western; Cells, Cultured; Dose-Response Relationship, Dru | 2003 |
Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy.
Topics: Animals; Antibodies, Monoclonal; Blotting, Western; Cells, Cultured; Dose-Response Relationship, Dru | 2003 |
Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy.
Topics: Animals; Antibodies, Monoclonal; Blotting, Western; Cells, Cultured; Dose-Response Relationship, Dru | 2003 |
Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy.
Topics: Animals; Antibodies, Monoclonal; Blotting, Western; Cells, Cultured; Dose-Response Relationship, Dru | 2003 |
Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy.
Topics: Animals; Antibodies, Monoclonal; Blotting, Western; Cells, Cultured; Dose-Response Relationship, Dru | 2003 |
Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy.
Topics: Animals; Antibodies, Monoclonal; Blotting, Western; Cells, Cultured; Dose-Response Relationship, Dru | 2003 |
Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy.
Topics: Animals; Antibodies, Monoclonal; Blotting, Western; Cells, Cultured; Dose-Response Relationship, Dru | 2003 |
Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy.
Topics: Animals; Antibodies, Monoclonal; Blotting, Western; Cells, Cultured; Dose-Response Relationship, Dru | 2003 |
Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy.
Topics: Animals; Antibodies, Monoclonal; Blotting, Western; Cells, Cultured; Dose-Response Relationship, Dru | 2003 |
Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy.
Topics: Animals; Antibodies, Monoclonal; Blotting, Western; Cells, Cultured; Dose-Response Relationship, Dru | 2003 |
Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy.
Topics: Animals; Antibodies, Monoclonal; Blotting, Western; Cells, Cultured; Dose-Response Relationship, Dru | 2003 |
Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy.
Topics: Animals; Antibodies, Monoclonal; Blotting, Western; Cells, Cultured; Dose-Response Relationship, Dru | 2003 |
The role of histone acetylation in SMN gene expression.
Topics: Acetylation; Animals; Cells, Cultured; Chromatin Immunoprecipitation; Cyclic AMP Response Element-Bi | 2005 |
The role of histone acetylation in SMN gene expression.
Topics: Acetylation; Animals; Cells, Cultured; Chromatin Immunoprecipitation; Cyclic AMP Response Element-Bi | 2005 |
Valproic acid promotes neurite outgrowth in PC12 cells independent from regulation of the survival of motoneuron protein.
Topics: Animals; Anticonvulsants; Cell Differentiation; Cell Enlargement; Cyclic AMP Response Element-Bindin | 2006 |
Valproic acid promotes neurite outgrowth in PC12 cells independent from regulation of the survival of motoneuron protein.
Topics: Animals; Anticonvulsants; Cell Differentiation; Cell Enlargement; Cyclic AMP Response Element-Bindin | 2006 |
Valproate may improve strength and function in patients with type III/IV spinal muscle atrophy.
Topics: Adolescent; Adult; Female; Humans; Male; Middle Aged; Motor Neurons; Muscular Atrophy, Spinal; Retro | 2006 |
Valproate may improve strength and function in patients with type III/IV spinal muscle atrophy.
Topics: Adolescent; Adult; Female; Humans; Male; Middle Aged; Motor Neurons; Muscular Atrophy, Spinal; Retro | 2006 |
Establishing a standardized therapeutic testing protocol for spinal muscular atrophy.
Topics: Animals; Cell Death; Cell Survival; Cyclic AMP Response Element-Binding Protein; Disease Models, Ani | 2006 |
Establishing a standardized therapeutic testing protocol for spinal muscular atrophy.
Topics: Animals; Cell Death; Cell Survival; Cyclic AMP Response Element-Binding Protein; Disease Models, Ani | 2006 |
Valproic acid treatment in six patients with spinal muscular atrophy.
Topics: Adolescent; Adult; Age Factors; Anticonvulsants; Child; Child, Preschool; Dose-Response Relationship | 2007 |
Valproic acid treatment in six patients with spinal muscular atrophy.
Topics: Adolescent; Adult; Age Factors; Anticonvulsants; Child; Child, Preschool; Dose-Response Relationship | 2007 |