Bigger BW - Search Results

1

Improving newborn screening test performance for metachromatic leukodystrophy: Recommendation from a pre-pilot study that identified a late-infantile case for treatment.

Wu THY, Brown HA, Church HJ, Kershaw CJ, Hutton R, Egerton C, Cooper J, Tylee K, Cohen RN, Gokhale D, Ram D, Morton G, Henderson M, Bigger BW, Jones SA. Wu THY, et al. Among authors: bigger bw. Mol Genet Metab. 2024 May;142(1):108349. doi: 10.1016/j.ymgme.2024.108349. Epub 2024 Feb 20. Mol Genet Metab. 2024. PMID: 38458124 Free article.

2

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: bigger bw. 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

3

Enzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10 year combined experience of 2 centres.

Ghosh A, Miller W, Orchard PJ, Jones SA, Mercer J, Church HJ, Tylee K, Lund T, Bigger BW, Tolar J, Wynn RF. Ghosh A, et al. Among authors: bigger bw. Mol Genet Metab. 2016 Mar;117(3):373-7. doi: 10.1016/j.ymgme.2016.01.011. Epub 2016 Jan 27. Mol Genet Metab. 2016. PMID: 26832957 Free PMC article.

4

Current and Future Treatment of Mucopolysaccharidosis (MPS) Type II: Is Brain-Targeted Stem Cell Gene Therapy the Solution for This Devastating Disorder?

Horgan C, Jones SA, Bigger BW, Wynn R. Horgan C, et al. Among authors: bigger bw. Int J Mol Sci. 2022 Apr 27;23(9):4854. doi: 10.3390/ijms23094854. Int J Mol Sci. 2022. PMID: 35563245 Free PMC article. Review.

5

Sleep disordered breathing in mucopolysaccharidosis I: a multivariate analysis of patient, therapeutic and metabolic correlators modifying long term clinical outcome.

Pal AR, Langereis EJ, Saif MA, Mercer J, Church HJ, Tylee KL, Wynn RF, Wijburg FA, Jones SA, Bruce IA, Bigger BW. Pal AR, et al. Among authors: bigger bw. Orphanet J Rare Dis. 2015 Apr 10;10:42. doi: 10.1186/s13023-015-0255-4. Orphanet J Rare Dis. 2015. PMID: 25887468 Free PMC article.

6

Improved metabolic correction in patients with lysosomal storage disease treated with hematopoietic stem cell transplant compared with enzyme replacement therapy.

Wynn RF, Wraith JE, Mercer J, O'Meara A, Tylee K, Thornley M, Church HJ, Bigger BW. Wynn RF, et al. Among authors: bigger bw. J Pediatr. 2009 Apr;154(4):609-11. doi: 10.1016/j.jpeds.2008.11.005. J Pediatr. 2009. PMID: 19324223 Clinical Trial.

7

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: bigger bw. 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.

8

Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler's syndrome after pharmacological enzyme replacement therapy.

Saif MA, Bigger BW, Brookes KE, Mercer J, Tylee KL, Church HJ, Bonney DK, Jones S, Wraith JE, Wynn RF. Saif MA, et al. Among authors: bigger bw. Haematologica. 2012 Sep;97(9):1320-8. doi: 10.3324/haematol.2011.058644. Epub 2012 Feb 27. Haematologica. 2012. PMID: 22371174 Free PMC article.

9

Substrate accumulation and extracellular matrix remodelling promote persistent upper airway disease in mucopolysaccharidosis patients on enzyme replacement therapy.

Pal AR, Mercer J, Jones SA, Bruce IA, Bigger BW. Pal AR, et al. Among authors: bigger bw. PLoS One. 2018 Sep 18;13(9):e0203216. doi: 10.1371/journal.pone.0203216. eCollection 2018. PLoS One. 2018. PMID: 30226843 Free PMC article.

10

Strategies for the Induction of Immune Tolerance to Enzyme Replacement Therapy in Mucopolysaccharidosis Type I.

Ghosh A, Liao A, O'Leary C, Mercer J, Tylee K, Goenka A, Holley R, Jones SA, Bigger BW. Ghosh A, et al. Among authors: bigger bw. Mol Ther Methods Clin Dev. 2019 Mar 2;13:321-333. doi: 10.1016/j.omtm.2019.02.007. eCollection 2019 Jun 14. Mol Ther Methods Clin Dev. 2019. PMID: 30976609 Free PMC article.

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