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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: maczulski ja. 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.
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: maczulski ja. 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: maczulski ja. PLoS One. 2010 Aug 19;5(8):e12140. doi: 10.1371/journal.pone.0012140. PLoS One. 2010. PMID: 20808854 Free PMC article. Clinical Trial.
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. Finkel RS, et al. PLoS One. 2012;7(4):e35462. doi: 10.1371/journal.pone.0035462. Epub 2012 Apr 27. PLoS One. 2012. PMID: 22558154 Free PMC article. Clinical Trial.
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: maczulski ja. Muscle Nerve. 2014 Feb;49(2):187-92. doi: 10.1002/mus.23904. Muscle Nerve. 2014. PMID: 23681940 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. Among authors: maczulski ja. 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.