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Page 1
Parent-reported benefits of flupirtine in juvenile neuronal ceroid lipofuscinosis (Batten disease; CLN3) are not supported by quantitative data.
Cialone J, Augustine EF, Newhouse N, Adams H, Vierhile A, Marshall FJ, de Blieck EA, Kwon J, Rothberg PG, Mink JW. Cialone J, et al. Among authors: de blieck ea. J Inherit Metab Dis. 2011 Oct;34(5):1075-81. doi: 10.1007/s10545-011-9346-0. Epub 2011 May 10. J Inherit Metab Dis. 2011. PMID: 21556831 Free PMC article.
Females experience a more severe disease course in Batten disease.
Cialone J, Adams H, Augustine EF, Marshall FJ, Kwon JM, Newhouse N, Vierhile A, Levy E, Dure LS, Rose KR, Ramirez-Montealegre D, de Blieck EA, Mink JW. Cialone J, et al. Among authors: de blieck ea. J Inherit Metab Dis. 2012 May;35(3):549-55. doi: 10.1007/s10545-011-9421-6. Epub 2011 Dec 14. J Inherit Metab Dis. 2012. PMID: 22167274 Free PMC article.
A clinical rating scale for Batten disease: reliable and relevant for clinical trials.
Marshall FJ, de Blieck EA, Mink JW, Dure L, Adams H, Messing S, Rothberg PG, Levy E, McDonough T, DeYoung J, Wang M, Ramirez-Montealegre D, Kwon JM, Pearce DA. Marshall FJ, et al. Among authors: de blieck ea. Neurology. 2005 Jul 26;65(2):275-9. doi: 10.1212/01.wnl.0000169019.41332.8a. Neurology. 2005. PMID: 16043799
Quantifying physical decline in juvenile neuronal ceroid lipofuscinosis (Batten disease).
Kwon JM, Adams H, Rothberg PG, Augustine EF, Marshall FJ, Deblieck EA, Vierhile A, Beck CA, Newhouse NJ, Cialone J, Levy E, Ramirez-Montealegre D, Dure LS, Rose KR, Mink JW. Kwon JM, et al. Neurology. 2011 Nov 15;77(20):1801-7. doi: 10.1212/WNL.0b013e318237f649. Epub 2011 Oct 19. Neurology. 2011. PMID: 22013180 Free PMC article.
Methodology of clinical research in rare diseases: development of a research program in juvenile neuronal ceroid lipofuscinosis (JNCL) via creation of a patient registry and collaboration with patient advocates.
de Blieck EA, Augustine EF, Marshall FJ, Adams H, Cialone J, Dure L, Kwon JM, Newhouse N, Rose K, Rothberg PG, Vierhile A, Mink JW; Batten Study Group. de Blieck EA, et al. Contemp Clin Trials. 2013 Jul;35(2):48-54. doi: 10.1016/j.cct.2013.04.004. Epub 2013 Apr 26. Contemp Clin Trials. 2013. PMID: 23628560 Free PMC article.
A futility study of minocycline in Huntington's disease.
Huntington Study Group DOMINO Investigators. Huntington Study Group DOMINO Investigators. Mov Disord. 2010 Oct 15;25(13):2219-24. doi: 10.1002/mds.23236. Mov Disord. 2010. PMID: 20721920 Free PMC article. Clinical Trial.
27 results