Publications

Name/Description
Iyer CC, Corlett KM, Massoni-Laporte A, Duque SI, Madabusi N, Tisdale S, McGovern VL, Le TT, Zaworski PG, David Arnold W, Pellizzoni L, Burghes AHM. 2018. Mild SMN missense alleles are only functional in the presence of SMN2 in mammals. Hum Mol Genet 2018 Jul 6. doi: 10.1093/hmg/ddy251. [Epub ahead of print]
Kolb SJ, Coffey CS, Yankey JW, Krosschell K, Arnold WD, Rutkove SB, Swoboda KJ, Reyna SP, Sakonju A, Darras BT, Shell R, Kuntz N, Castro D, Parsons J, Connolly AM, Chiriboga CA, McDonald C, Burnette WB, Werner K, Thangarajh M, Shieh PB, Finanger E, Cudkowicz ME, McGovern MM, McNeil DE, Finkel R, Iannaccone ST, Kaye E, Kingsley A, Renusch SR, McGovern VL, Wang X, Zaworski PG, Prior TW, Burghes AHM, Bartlett A, Kissel JT; NeuroNEXT Clinical Trial Network on behalf of the NN101 SMA Biomarker Investigators. 2017. Natural history of infantile-onset spinal muscular atrophy. Ann Neurol 82(6):883-891.
Arnold WD, Duque S, Iyer CC, Zaworski P, McGovern VL, Taylor SJ, von Herrmann KM, Kobayashi DT, Chen KS, Kolb SJ, Paushkin SV, Burghes AH. 2016. Normalization of Patient-Identified Plasma Biomarkers in SMNΔ7 Mice following Postnatal SMN Restoration. PLoS One 11(12):e0167077. doi: 10.1371/journal.pone.0167077. eCollection 2016.
Zaworski P, von Herrmann KM, Taylor S, Sunshine SS, McCarthy K, Risher N, Newcomb T, Weetall M, Prior TW, Swoboda KJ, Chen KS, Paushkin S. 2016. SMN Protein Can Be Reliably Measured in Whole Blood with an Electrochemiluminescence (ECL) Immunoassay: Implications for Clinical Trials. PLoS One 11(3):e0150640. doi: 10.1371/journal.pone.0150640. eCollection 2016.
Kolb SJ, Coffey CS, Yankey JW, Krosschell K, Arnold WD, Rutkove SB, Swoboda KJ, Reyna SP, Sakonju A, Darras BT, Shell R, Kuntz N, Castro D, Iannaccone ST, Parsons J, Connolly AM, Chiriboga CA, McDonald C, Burnette WB, Werner K, Thangarajh M, Shieh PB, Finanger E, Cudkowicz ME, McGovern MM, McNeil DE, Finkel R, Kaye E, Kingsley A, Renusch SR, McGovern VL, Wang X, Zaworski PG, Prior TW, Burghes AH, Bartlett A, Kissel JT; NeuroNEXT Clinical Trial Network and on behalf of the NN101 SMA Biomarker Investigators. 2016. Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study. Ann Clin Transl Neurol 3(2):132-45. doi: 10.1002/acn3.283. eCollection 2016 Feb.
Czech C, Tang W, Bugawan T, Mano C, Horn C, Iglesias VA, Fröhner S, Zaworski PG, Paushkin S, Chen K, Kremer T. 2015. Biomarker for Spinal Muscular Atrophy: Expression of SMN in Peripheral Blood of SMA Patients and Healthy Controls. PLoS One 10(10):e0139950. doi: 10.1371/journal.pone.0139950. eCollection 2015.
Iyer CC, McGovern VL, Murray JD, Gombash SE, Zaworski PG, Foust KD, Janssen PM, Burghes AH. 2015. Low levels of Survival Motor Neuron protein are sufficient for normal muscle function in the SMNΔ7 mouse model of SMA. Hum Mol Genet 24(21):6160-73. doi: 10.1093/hmg/ddv332. Epub 2015 Aug 13
McGovern VL, Iyer CC, Arnold WD, Gombash SE, Zaworski PG, Blatnik AJ 3rd, Foust KD, Burghes AH. 2015. SMN expression is required in motor neurons to rescue electrophysiological deficits in the SMNΔ7 mouse model of SMA. Hum Mol Genet 24(19):5524-41. doi: 10.1093/hmg/ddv283. Epub 2015 Jul 23.
Li J, Geisbush TR, Arnold WD, Rosen GD, Zaworski PG, Rutkove SB. 2014. A comparison of three electrophysiological methods for the assessment of disease status in a mild spinal muscular atrophy mouse model. PLoS One 9(10):e111428. doi: 10.1371/journal.pone.0111428. eCollection 2014.
Cherry JJ, Kobayashi DT, Lynes MM, Naryshkin NN, Tiziano FD, Zaworski PG, Rubin LL, Jarecki J. Assays for the identification and prioritization of drug candidates for spinal muscular atrophy. 2014. • Assay Drug Dev Technol 12(6):315-41. doi: 10.1089/adt.2014.587.
Kobayashi DT, Decker D, Zaworski P, Klott K, McGonigal J, Ghazal N, Sly L, Chung B, Vanderlugt J, Chen KS. 2012. Evaluation of peripheral blood mononuclear cell processing and analysis for Survival Motor Neuron protein. PLoS One 7(11):e50763. doi: 10.1371/journal.pone.0050763. Epub 2012 Nov 30.


Sponsor publication with acknowledgment that Pharmacokinetic studies were completed by PharmOptima:
Schopf L, Enlow E, Popov A, Bourassa J, Chen H. 2014. Ocular Pharmacokinetics of Novel Loteprednol Etabonate 0.4% Opthalmic Formulation. Ophthalmol Ther 3(1-2): 63-72.