Thurberg BL - Search Results

1

Improved muscle function in a phase I/II clinical trial of albuterol in Pompe disease.

Koeberl DD, Case LE, Desai A, Smith EC, Walters C, Han SO, Thurberg BL, Young SP, Bali D, Kishnani PS. Koeberl DD, et al. Among authors: thurberg bl. Mol Genet Metab. 2020 Feb;129(2):67-72. doi: 10.1016/j.ymgme.2019.12.008. Epub 2019 Dec 10. Mol Genet Metab. 2020. PMID: 31839530 Clinical Trial.

2

Enzyme replacement therapy in the mouse model of Pompe disease.

Raben N, Danon M, Gilbert AL, Dwivedi S, Collins B, Thurberg BL, Mattaliano RJ, Nagaraju K, Plotz PH. Raben N, et al. Among authors: thurberg bl. Mol Genet Metab. 2003 Sep-Oct;80(1-2):159-69. doi: 10.1016/j.ymgme.2003.08.022. Mol Genet Metab. 2003. PMID: 14567965

3

Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease.

Kishnani PS, Nicolino M, Voit T, Rogers RC, Tsai AC, Waterson J, Herman GE, Amalfitano A, Thurberg BL, Richards S, Davison M, Corzo D, Chen YT. Kishnani PS, et al. Among authors: thurberg bl. J Pediatr. 2006 Jul;149(1):89-97. doi: 10.1016/j.jpeds.2006.02.035. J Pediatr. 2006. PMID: 16860134 Free PMC article. Clinical Trial.

4

Characterization of pre- and post-treatment pathology after enzyme replacement therapy for Pompe disease.

Thurberg BL, Lynch Maloney C, Vaccaro C, Afonso K, Tsai AC, Bossen E, Kishnani PS, O'Callaghan M. Thurberg BL, et al. Lab Invest. 2006 Dec;86(12):1208-20. doi: 10.1038/labinvest.3700484. Epub 2006 Oct 30. Lab Invest. 2006. PMID: 17075580 Free article.

5

Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease.

Kishnani PS, Corzo D, Nicolino M, Byrne B, Mandel H, Hwu WL, Leslie N, Levine J, Spencer C, McDonald M, Li J, Dumontier J, Halberthal M, Chien YH, Hopkin R, Vijayaraghavan S, Gruskin D, Bartholomew D, van der Ploeg A, Clancy JP, Parini R, Morin G, Beck M, De la Gastine GS, Jokic M, Thurberg B, Richards S, Bali D, Davison M, Worden MA, Chen YT, Wraith JE. Kishnani PS, et al. Neurology. 2007 Jan 9;68(2):99-109. doi: 10.1212/01.wnl.0000251268.41188.04. Epub 2006 Dec 6. Neurology. 2007. PMID: 17151339 Clinical Trial.

6

Differential muscular glycogen clearance after enzyme replacement therapy in a mouse model of Pompe disease.

Hawes ML, Kennedy W, O'Callaghan MW, Thurberg BL. Hawes ML, et al. Among authors: thurberg bl. Mol Genet Metab. 2007 Aug;91(4):343-51. doi: 10.1016/j.ymgme.2007.04.018. Epub 2007 Jun 14. Mol Genet Metab. 2007. PMID: 17572127

7

Biochemical and pharmacological characterization of different recombinant acid alpha-glucosidase preparations evaluated for the treatment of Pompe disease.

McVie-Wylie AJ, Lee KL, Qiu H, Jin X, Do H, Gotschall R, Thurberg BL, Rogers C, Raben N, O'Callaghan M, Canfield W, Andrews L, McPherson JM, Mattaliano RJ. McVie-Wylie AJ, et al. Among authors: thurberg bl. Mol Genet Metab. 2008 Aug;94(4):448-455. doi: 10.1016/j.ymgme.2008.04.009. Epub 2008 Jun 5. Mol Genet Metab. 2008. PMID: 18538603 Free PMC article.

8

Long-term monitoring of patients with infantile-onset Pompe disease on enzyme replacement therapy using a urinary glucose tetrasaccharide biomarker.

Young SP, Zhang H, Corzo D, Thurberg BL, Bali D, Kishnani PS, Millington DS. Young SP, et al. Among authors: thurberg bl. Genet Med. 2009 Jul;11(7):536-41. doi: 10.1097/GIM.0b013e3181a87867. Genet Med. 2009. PMID: 19521244 Free article. Clinical Trial.

9

Antibody formation and mannose-6-phosphate receptor expression impact the efficacy of muscle-specific transgene expression in murine Pompe disease.

Sun B, Li S, Bird A, Yi H, Kemper A, Thurberg BL, Koeberl DD. Sun B, et al. Among authors: thurberg bl. J Gene Med. 2010 Nov;12(11):881-91. doi: 10.1002/jgm.1511. Epub 2010 Oct 22. J Gene Med. 2010. PMID: 20967919 Free PMC article.

10

Expanding the clinical spectrum of late-onset Pompe disease: dilated arteriopathy involving the thoracic aorta, a novel vascular phenotype uncovered.

El-Gharbawy AH, Bhat G, Murillo JE, Thurberg BL, Kampmann C, Mengel KE, Kishnani PS. El-Gharbawy AH, et al. Among authors: thurberg bl. Mol Genet Metab. 2011 Aug;103(4):362-6. doi: 10.1016/j.ymgme.2011.04.009. Epub 2011 May 5. Mol Genet Metab. 2011. PMID: 21605996

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