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Items: 1 to 20 of 109

1.

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

Swoboda KJ, Scott CB, Crawford TO, Simard LR, Reyna SP, Krosschell KJ, Acsadi G, Elsheik B, Schroth MK, D'Anjou G, LaSalle B, Prior TW, Sorenson SL, Maczulski JA, Bromberg MB, Chan GM, Kissel JT; Project Cure Spinal Muscular Atrophy Investigators Network.

PLoS One. 2010 Aug 19;5(8):e12140. doi: 10.1371/journal.pone.0012140.

2.

SMA CARNIVAL TRIAL PART II: a prospective, single-armed trial of L-carnitine and valproic acid in ambulatory children with spinal muscular atrophy.

Kissel JT, Scott CB, Reyna SP, Crawford TO, Simard LR, Krosschell KJ, Acsadi G, Elsheik B, Schroth MK, D'Anjou G, LaSalle B, Prior TW, Sorenson S, Maczulski JA, Bromberg MB, Chan GM, Swoboda KJ; Project Cure Spinal Muscular Atrophy Investigators' Network.

PLoS One. 2011;6(7):e21296. doi: 10.1371/journal.pone.0021296. Epub 2011 Jul 6.

3.

Phase II open label study of valproic acid in spinal muscular atrophy.

Swoboda KJ, Scott CB, Reyna SP, Prior TW, LaSalle B, Sorenson SL, Wood J, Acsadi G, Crawford TO, Kissel JT, Krosschell KJ, D'Anjou G, Bromberg MB, Schroth MK, Chan GM, Elsheikh B, Simard LR.

PLoS One. 2009;4(5):e5268. doi: 10.1371/journal.pone.0005268. Epub 2009 May 14.

4.

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

Kissel JT, Elsheikh B, King WM, Freimer M, Scott CB, Kolb SJ, Reyna SP, Crawford TO, Simard LR, Krosschell KJ, Acsadi G, Schroth MK, D'Anjou G, LaSalle B, Prior TW, Sorenson S, Maczulski JA, Swoboda KJ; Project Cure Spinal Muscular Atrophy Investigators Network.

Muscle Nerve. 2014 Feb;49(2):187-92.

5.

Randomized, double-blind, placebo-controlled trial of hydroxyurea in spinal muscular atrophy.

Chen TH, Chang JG, Yang YH, Mai HH, Liang WC, Wu YC, Wang HY, Huang YB, Wu SM, Chen YC, Yang SN, Jong YJ.

Neurology. 2010 Dec 14;75(24):2190-7. doi: 10.1212/WNL.0b013e3182020332.

PMID:
21172842
6.

A phase 1 trial of riluzole in spinal muscular atrophy.

Russman BS, Iannaccone ST, Samaha FJ.

Arch Neurol. 2003 Nov;60(11):1601-3.

PMID:
14623733
7.

Multiple therapeutic effects of valproic acid in spinal muscular atrophy model mice.

Tsai LK, Tsai MS, Ting CH, Li H.

J Mol Med (Berl). 2008 Nov;86(11):1243-54. doi: 10.1007/s00109-008-0388-1. Epub 2008 Jul 23.

PMID:
18649067
8.

Compound muscle action potential and motor function in children with spinal muscular atrophy.

Lewelt A, Krosschell KJ, Scott C, Sakonju A, Kissel JT, Crawford TO, Acsadi G, D'anjou G, Elsheikh B, Reyna SP, Schroth MK, Maczulski JA, Stoddard GJ, Elovic E, Swoboda KJ.

Muscle Nerve. 2010 Nov;42(5):703-8. doi: 10.1002/mus.21838.

9.

The effect of hydroxyurea in spinal muscular atrophy cells and patients.

Liang WC, Yuo CY, Chang JG, Chen YC, Chang YF, Wang HY, Ju YH, Chiou SS, Jong YJ.

J Neurol Sci. 2008 May 15;268(1-2):87-94. doi: 10.1016/j.jns.2007.11.012. Epub 2007 Dec 31.

PMID:
18166199
10.

Randomized, double-blind, placebo-controlled trial of phenylbutyrate in spinal muscular atrophy.

Mercuri E, Bertini E, Messina S, Solari A, D'Amico A, Angelozzi C, Battini R, Berardinelli A, Boffi P, Bruno C, Cini C, Colitto F, Kinali M, Minetti C, Mongini T, Morandi L, Neri G, Orcesi S, Pane M, Pelliccioni M, Pini A, Tiziano FD, Villanova M, Vita G, Brahe C.

Neurology. 2007 Jan 2;68(1):51-5. Epub 2006 Nov 2.

PMID:
17082463
11.

Evaluation of SMN protein, transcript, and copy number in the biomarkers for spinal muscular atrophy (BforSMA) clinical study.

Crawford TO, Paushkin SV, Kobayashi DT, Forrest SJ, Joyce CL, Finkel RS, Kaufmann P, Swoboda KJ, Tiziano D, Lomastro R, Li RH, Trachtenberg FL, Plasterer T, Chen KS; Pilot Study of Biomarkers for Spinal Muscular Atrophy Trial Group.

PLoS One. 2012;7(4):e33572. doi: 10.1371/journal.pone.0033572. Epub 2012 Apr 27.

12.

Candidate proteins, metabolites and transcripts in the Biomarkers for Spinal Muscular Atrophy (BforSMA) clinical study.

Finkel RS, Crawford TO, Swoboda KJ, Kaufmann P, Juhasz P, Li X, Guo Y, Li RH, Trachtenberg F, Forrest SJ, Kobayashi DT, Chen KS, Joyce CL, Plasterer T; Pilot Study of Biomarkers for Spinal Muscular Atrophy Trial Group.

PLoS One. 2012;7(4):e35462. doi: 10.1371/journal.pone.0035462. Epub 2012 Apr 27.

13.

Salbutamol increases survival motor neuron (SMN) transcript levels in leucocytes of spinal muscular atrophy (SMA) patients: relevance for clinical trial design.

Tiziano FD, Lomastro R, Pinto AM, Messina S, D'Amico A, Fiori S, Angelozzi C, Pane M, Mercuri E, Bertini E, Neri G, Brahe C.

J Med Genet. 2010 Dec;47(12):856-8. doi: 10.1136/jmg.2010.080366. Epub 2010 Sep 12.

PMID:
20837492
14.

Evaluation of muscle strength and motor abilities in children with type II and III spinal muscle atrophy treated with valproic acid.

Darbar IA, Plaggert PG, Resende MB, Zanoteli E, Reed UC.

BMC Neurol. 2011 Mar 24;11:36. doi: 10.1186/1471-2377-11-36.

15.

Mitoxantrone: a review of its use in multiple sclerosis.

Scott LJ, Figgitt DP.

CNS Drugs. 2004;18(6):379-96. Review.

PMID:
15089110
16.

Quantification of SMN protein in leucocytes from spinal muscular atrophy patients: effects of treatment with valproic acid.

Piepers S, Cobben JM, Sodaar P, Jansen MD, Wadman RI, Meester-Delver A, Poll-The BT, Lemmink HH, Wokke JH, van der Pol WL, van den Berg LH.

J Neurol Neurosurg Psychiatry. 2011 Aug;82(8):850-2. doi: 10.1136/jnnp.2009.200253. Epub 2010 Jun 15.

PMID:
20551479
17.

Responsiveness of the motor function measure in patients with spinal muscular atrophy.

Vuillerot C, Payan C, Iwaz J, Ecochard R, BĂ©rard C; MFM Spinal Muscular Atrophy Study Group.

Arch Phys Med Rehabil. 2013 Aug;94(8):1555-61. doi: 10.1016/j.apmr.2013.01.014. Epub 2013 Feb 1.

PMID:
23380348
19.

Efficacy and safety of leuprorelin in patients with spinal and bulbar muscular atrophy (JASMITT study): a multicentre, randomised, double-blind, placebo-controlled trial.

Katsuno M, Banno H, Suzuki K, Takeuchi Y, Kawashima M, Yabe I, Sasaki H, Aoki M, Morita M, Nakano I, Kanai K, Ito S, Ishikawa K, Mizusawa H, Yamamoto T, Tsuji S, Hasegawa K, Shimohata T, Nishizawa M, Miyajima H, Kanda F, Watanabe Y, Nakashima K, Tsujino A, Yamashita T, Uchino M, Fujimoto Y, Tanaka F, Sobue G; Japan SBMA Interventional Trial for TAP-144-SR (JASMITT) study group.

Lancet Neurol. 2010 Sep;9(9):875-84. doi: 10.1016/S1474-4422(10)70182-4. Epub 2010 Aug 4.

PMID:
20691641
20.

A placebo-controlled screening trial of olanzapine, valproate, and coenzyme Q10/L-carnitine for the treatment of cocaine dependence.

Reid MS, Casadonte P, Baker S, Sanfilipo M, Braunstein D, Hitzemann R, Montgomery A, Majewska D, Robinson J, Rotrosen J.

Addiction. 2005 Mar;100 Suppl 1:43-57.

PMID:
15730349
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