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Immunosuppression alters disease severity in juvenile Batten disease mice.
Seehafer SS, Ramirez-Montealegre D, Wong AM, Chan CH, Castaneda J, Horak M, Ahmadi SM, Lim MJ, Cooper JD, Pearce DA. Seehafer SS, et al. J Neuroimmunol. 2011 Jan;230(1-2):169-72. doi: 10.1016/j.jneuroim.2010.08.024. J Neuroimmunol. 2011. PMID: 20937531 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.