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Clinical trial of L-Carnitine and valproic acid in spinal muscular atrophy type I.
Krosschell KJ, Kissel JT, Townsend EL, Simeone SD, Zhang RZ, Reyna SP, Crawford TO, Schroth MK, Acsadi G, Kishnani PS, Von Kleist-Retzow JC, Hero B, D'Anjou G, Smith EC, Elsheikh B, Simard LR, Prior TW, Scott CB, Lasalle B, Sakonju A, Wirth B, Swoboda KJ; Project Cure SMA Investigator's Network. Krosschell KJ, et al. Among authors: acsadi g. Muscle Nerve. 2018 Feb;57(2):193-199. doi: 10.1002/mus.25776. Epub 2017 Sep 18. Muscle Nerve. 2018. PMID: 28833236 Clinical Trial.
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. Lewelt A, et al. Among authors: acsadi g. Muscle Nerve. 2010 Nov;42(5):703-8. doi: 10.1002/mus.21838. Muscle Nerve. 2010. PMID: 20737553 Free PMC article. Clinical Trial.
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. Swoboda KJ, et al. Among authors: acsadi g. PLoS One. 2010 Aug 19;5(8):e12140. doi: 10.1371/journal.pone.0012140. PLoS One. 2010. PMID: 20808854 Free PMC article. Clinical Trial.
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. Kissel JT, et al. Among authors: acsadi g. PLoS One. 2011;6(7):e21296. doi: 10.1371/journal.pone.0021296. Epub 2011 Jul 6. PLoS One. 2011. PMID: 21754985 Free PMC article. Clinical Trial.
Reliability and validity of the TIMPSI for infants with spinal muscular atrophy type I.
Krosschell KJ, Maczulski JA, Scott C, King W, Hartman JT, Case LE, Viazzo-Trussell D, Wood J, Roman CA, Hecker E, Meffert M, Léveillé M, Kienitz K, Swoboda KJ; Project Cure SMA Investigators Network. Krosschell KJ, et al. Pediatr Phys Ther. 2013 Summer;25(2):140-8; discussion 149. doi: 10.1097/PEP.0b013e31828a205f. Pediatr Phys Ther. 2013. PMID: 23542189 Free PMC article.
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. Kissel JT, et al. Among authors: acsadi g. Muscle Nerve. 2014 Feb;49(2):187-92. doi: 10.1002/mus.23904. Muscle Nerve. 2014. PMID: 23681940 Free PMC article. Clinical Trial.
Safety and efficacy of nusinersen in spinal muscular atrophy: The EMBRACE study.
Acsadi G, Crawford TO, Müller-Felber W, Shieh PB, Richardson R, Natarajan N, Castro D, Ramirez-Schrempp D, Gambino G, Sun P, Farwell W. Acsadi G, et al. Muscle Nerve. 2021 May;63(5):668-677. doi: 10.1002/mus.27187. Epub 2021 Feb 16. Muscle Nerve. 2021. PMID: 33501671 Free PMC article. Clinical Trial.
Perspectives on clinical trials in spinal muscular atrophy.
Swoboda KJ, Kissel JT, Crawford TO, Bromberg MB, Acsadi G, D'Anjou G, Krosschell KJ, Reyna SP, Schroth MK, Scott CB, Simard LR. Swoboda KJ, et al. Among authors: acsadi g. J Child Neurol. 2007 Aug;22(8):957-66. doi: 10.1177/0883073807305665. J Child Neurol. 2007. PMID: 17761650 Free PMC article. Review.
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. Swoboda KJ, et al. Among authors: acsadi g. PLoS One. 2009;4(5):e5268. doi: 10.1371/journal.pone.0005268. Epub 2009 May 14. PLoS One. 2009. PMID: 19440247 Free PMC article. Clinical Trial.
Alpha-synuclein loss in spinal muscular atrophy.
Acsadi G, Li X, Murphy KJ, Swoboda KJ, Parker GC. Acsadi G, et al. J Mol Neurosci. 2011 Mar;43(3):275-83. doi: 10.1007/s12031-010-9422-1. Epub 2010 Jul 17. J Mol Neurosci. 2011. PMID: 20640532 Free PMC article.
85 results