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Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman disease.
Potter JE, Petts G, Ghosh A, White FJ, Kinsella JL, Hughes S, Roberts J, Hodgkinson A, Brammeier K, Church H, Merrigan C, Hughes J, Evans P, Campbell H, Bonney D, Newman WG, Bigger BW, Broomfield A, Jones SA, Wynn RF. Potter JE, et al. Among authors: church h. Orphanet J Rare Dis. 2021 May 21;16(1):235. doi: 10.1186/s13023-021-01849-7. Orphanet J Rare Dis. 2021. PMID: 34020687 Free PMC article.
Heparin cofactor II-thrombin complex and dermatan sulphate:chondroitin sulphate ratio are biomarkers of short- and long-term treatment effects in mucopolysaccharide diseases.
Langford-Smith KJ, Mercer J, Petty J, Tylee K, Church H, Roberts J, Moss G, Jones S, Wynn R, Wraith JE, Bigger BW. Langford-Smith KJ, et al. Among authors: church h. J Inherit Metab Dis. 2011 Apr;34(2):499-508. doi: 10.1007/s10545-010-9254-8. Epub 2010 Dec 18. J Inherit Metab Dis. 2011. PMID: 21170681 Free PMC article.
Biomarker responses correlate with antibody status in mucopolysaccharidosis type I patients on long-term enzyme replacement therapy.
Langereis EJ, van Vlies N, Church HJ, Geskus RB, Hollak CE, Jones SA, Kulik W, van Lenthe H, Mercer J, Schreider L, Tylee KL, Wagemans T, Wijburg FA, Bigger BW. Langereis EJ, et al. Among authors: church hj. Mol Genet Metab. 2015 Feb;114(2):129-37. doi: 10.1016/j.ymgme.2014.10.012. Epub 2014 Oct 29. Mol Genet Metab. 2015. PMID: 25467058
118 results