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1,122 results

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Page 1
Substrate reduction therapy for Krabbe disease and metachromatic leukodystrophy using a novel ceramide galactosyltransferase inhibitor.
Babcock MC, Mikulka CR, Wang B, Chandriani S, Chandra S, Xu Y, Webster K, Feng Y, Nelvagal HR, Giaramita A, Yip BK, Lo M, Jiang X, Chao Q, Woloszynek JC, Shen Y, Bhagwat S, Sands MS, Crawford BE. Babcock MC, et al. Among authors: webster k. Sci Rep. 2021 Jul 14;11(1):14486. doi: 10.1038/s41598-021-93601-1. Sci Rep. 2021. PMID: 34262084 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: webster ka. 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.
Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB.
Kan SH, Aoyagi-Scharber M, Le SQ, Vincelette J, Ohmi K, Bullens S, Wendt DJ, Christianson TM, Tiger PM, Brown JR, Lawrence R, Yip BK, Holtzinger J, Bagri A, Crippen-Harmon D, Vondrak KN, Chen Z, Hague CM, Woloszynek JC, Cheung DS, Webster KA, Adintori EG, Lo MJ, Wong W, Fitzpatrick PA, LeBowitz JH, Crawford BE, Bunting S, Dickson PI, Neufeld EF. Kan SH, et al. Among authors: webster ka. Proc Natl Acad Sci U S A. 2014 Oct 14;111(41):14870-5. doi: 10.1073/pnas.1416660111. Epub 2014 Sep 29. Proc Natl Acad Sci U S A. 2014. PMID: 25267636 Free PMC article.
Evaluation of the safety, tolerability, pharmacokinetics and pharmacodynamics of SM17 in healthy volunteers: results from pre-clinical models and a first-in-human, randomized, double blinded clinical trial.
Xu G, Paglialunga S, Qian X, Ding R, Webster K, van Haarst A, Engel C, Hui CW, Lam LH, Li W, Wu WC, Rasmussen S, Hunt A, Leung SO. Xu G, et al. Among authors: webster k. Front Immunol. 2024 Dec 9;15:1495540. doi: 10.3389/fimmu.2024.1495540. eCollection 2024. Front Immunol. 2024. PMID: 39717777 Free PMC article. Clinical Trial.
Securing Commercial Nucleic Acid Synthesis.
Crawford FW, Webster K, Epstein GL, Roberts D, Fair J, Nevo S. Crawford FW, et al. Among authors: webster k. Rand Health Q. 2024 Dec 10;12(1):4. eCollection 2024 Dec. Rand Health Q. 2024. PMID: 39664983 Free PMC article.
Translation and cross-cultural adaptation to Spanish of the Anterior Cruciate Ligament Return to Sport after Injury Scale (SP ACL‑RSI): Measurement properties and responsiveness in a multisport sample.
Donat-Roca R, Sánchez-Socarrás V, Romero-Sánchez JM, Tárrega S, Estapé-Madinabeitia T, Escalona-Marfil C, Seijas R, Romero-Cullerés G, Ochoa C, Webster KE. Donat-Roca R, et al. Among authors: webster ke. J Exp Orthop. 2024 Nov 26;11(4):e70046. doi: 10.1002/jeo2.70046. eCollection 2024 Oct. J Exp Orthop. 2024. PMID: 39600427 Free PMC article.
1,122 results