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Tralesinidase Alfa Enzyme Replacement Therapy Prevents Disease Manifestations in a Canine Model of Mucopolysaccharidosis Type IIIB.
Ellinwood NM, Valentine BN, Hess AS, Jens JK, Snella EM, Jamil M, Hostetter SJ, Jeffery ND, Smith JD, Millman ST, Parsons RL, Butt MT, Chandra S, Egeland MT, Assis AB, Nelvagal HR, Cooper JD, Nestrasil I, Mueller BA, Labounek R, Paulson A, Prill H, Liu XY, Zhou H, Lawrence R, Crawford BE, Grover A, Cherala G, Melton AC, Cherukuri A, Vuillemenot BR, Wait JCM, O'Neill CA, Pinkstaff J, Kovalchin J, Zanelli E, McCullagh E. Ellinwood NM, et al. Among authors: wait jcm. J Pharmacol Exp Ther. 2022 Sep;382(3):277-286. doi: 10.1124/jpet.122.001119. Epub 2022 Jun 18. J Pharmacol Exp Ther. 2022. PMID: 35717448 Free PMC article.
Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B.
Grover A, Crippen-Harmon D, Nave L, Vincelette J, Wait JCM, Melton AC, Lawrence R, Brown JR, Webster KA, Yip BK, Baridon B, Vitelli C, Rigney S, Christianson TM, Tiger PMN, Lo MJ, Holtzinger J, Shaywitz AJ, Crawford BE, Fitzpatrick PA, LeBowitz JH, Bullens S, Aoyagi-Scharber M, Bunting S, O'Neill CA, Pinkstaff J, Bagri A. Grover A, et al. Among authors: wait jcm. Drug Deliv Transl Res. 2020 Apr;10(2):425-439. doi: 10.1007/s13346-019-00683-6. Drug Deliv Transl Res. 2020. PMID: 31942701 Free PMC article.