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Items: 1 to 20 of 218

1.

Teaching tolerance: New approaches to enzyme replacement therapy for Pompe disease.

Cousens LP, Mingozzi F, van der Marel S, Su Y, Garman R, Ferreira V, Martin W, Scott DW, De Groot AS.

Hum Vaccin Immunother. 2012 Oct;8(10):1459-64. doi: 10.4161/hv.21405. Epub 2012 Oct 1.

2.

A new assay for fast, reliable CRIM status determination in infantile-onset Pompe disease.

Wang Z, Okamoto P, Keutzer J.

Mol Genet Metab. 2014 Feb;111(2):92-100. doi: 10.1016/j.ymgme.2013.08.010. Epub 2013 Aug 29.

3.

Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease.

Messinger YH, Mendelsohn NJ, Rhead W, Dimmock D, Hershkovitz E, Champion M, Jones SA, Olson R, White A, Wells C, Bali D, Case LE, Young SP, Rosenberg AS, Kishnani PS.

Genet Med. 2012 Jan;14(1):135-42. doi: 10.1038/gim.2011.4.

4.

Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance.

Sun B, Bird A, Young SP, Kishnani PS, Chen YT, Koeberl DD.

Am J Hum Genet. 2007 Nov;81(5):1042-9. Epub 2007 Sep 21.

5.

B-Cell depletion and immunomodulation before initiation of enzyme replacement therapy blocks the immune response to acid alpha-glucosidase in infantile-onset Pompe disease.

Elder ME, Nayak S, Collins SW, Lawson LA, Kelley JS, Herzog RW, Modica RF, Lew J, Lawrence RM, Byrne BJ.

J Pediatr. 2013 Sep;163(3):847-54.e1. doi: 10.1016/j.jpeds.2013.03.002. Epub 2013 Apr 16.

6.

Predicting cross-reactive immunological material (CRIM) status in Pompe disease using GAA mutations: lessons learned from 10 years of clinical laboratory testing experience.

Bali DS, Goldstein JL, Banugaria S, Dai J, Mackey J, Rehder C, Kishnani PS.

Am J Med Genet C Semin Med Genet. 2012 Feb 15;160C(1):40-9. doi: 10.1002/ajmg.c.31319. Epub 2012 Jan 17.

7.

Mapping the T helper cell response to acid α-glucosidase in Pompe mice.

Nayak S, Sivakumar R, Cao O, Daniell H, Byrne BJ, Herzog RW.

Mol Genet Metab. 2012 Jun;106(2):189-95. doi: 10.1016/j.ymgme.2012.03.009. Epub 2012 Mar 23.

8.

CRIM-negative infantile Pompe disease: 42-month treatment outcome.

Rohrbach M, Klein A, Köhli-Wiesner A, Veraguth D, Scheer I, Balmer C, Lauener R, Baumgartner MR.

J Inherit Metab Dis. 2010 Dec;33(6):751-7. doi: 10.1007/s10545-010-9209-0. Epub 2010 Sep 30.

PMID:
20882352
9.

Oral delivery of Acid Alpha Glucosidase epitopes expressed in plant chloroplasts suppresses antibody formation in treatment of Pompe mice.

Su J, Sherman A, Doerfler PA, Byrne BJ, Herzog RW, Daniell H.

Plant Biotechnol J. 2015 Oct;13(8):1023-32. doi: 10.1111/pbi.12413. Epub 2015 Jun 5.

10.

CRIM-negative infantile Pompe disease: characterization of immune responses in patients treated with ERT monotherapy.

Berrier KL, Kazi ZB, Prater SN, Bali DS, Goldstein J, Stefanescu MC, Rehder CW, Botha EG, Ellaway C, Bhattacharya K, Tylki-Szymanska A, Karabul N, Rosenberg AS, Kishnani PS.

Genet Med. 2015 Nov;17(11):912-8. doi: 10.1038/gim.2015.6. Epub 2015 Mar 5. Erratum in: Genet Med. 2015 Jul;17(7):596. Rosenburg, Amy S [corrected to Rosenberg, Amy S].

11.

Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants.

Kishnani PS, Goldenberg PC, DeArmey SL, Heller J, Benjamin D, Young S, Bali D, Smith SA, Li JS, Mandel H, Koeberl D, Rosenberg A, Chen YT.

Mol Genet Metab. 2010 Jan;99(1):26-33. doi: 10.1016/j.ymgme.2009.08.003.

12.

Immunodominant liver-specific expression suppresses transgene-directed immune responses in murine pompe disease.

Zhang P, Sun B, Osada T, Rodriguiz R, Yang XY, Luo X, Kemper AR, Clay TM, Koeberl DD.

Hum Gene Ther. 2012 May;23(5):460-72. doi: 10.1089/hum.2011.063. Epub 2012 Mar 29.

13.

Partial phenotypic correction and immune tolerance induction to enzyme replacement therapy after hematopoietic stem cell gene transfer of alpha-glucosidase in Pompe disease.

Douillard-Guilloux G, Richard E, Batista L, Caillaud C.

J Gene Med. 2009 Apr;11(4):279-87. doi: 10.1002/jgm.1305.

PMID:
19263466
14.

Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease.

Sun B, Kulis MD, Young SP, Hobeika AC, Li S, Bird A, Zhang H, Li Y, Clay TM, Burks W, Kishnani PS, Koeberl DD.

Mol Ther. 2010 Feb;18(2):353-60. doi: 10.1038/mt.2009.195. Epub 2009 Aug 18.

15.

Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy.

Prater SN, Patel TT, Buckley AF, Mandel H, Vlodavski E, Banugaria SG, Feeney EJ, Raben N, Kishnani PS.

Orphanet J Rare Dis. 2013 Jun 20;8:90. doi: 10.1186/1750-1172-8-90.

16.

Immune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent.

Nayak S, Doerfler PA, Porvasnik SL, Cloutier DD, Khanna R, Valenzano KJ, Herzog RW, Byrne BJ.

PLoS One. 2014 Jun 4;9(6):e98336. doi: 10.1371/journal.pone.0098336. eCollection 2014.

17.

Enzyme therapy and immune response in relation to CRIM status: the Dutch experience in classic infantile Pompe disease.

van Gelder CM, Hoogeveen-Westerveld M, Kroos MA, Plug I, van der Ploeg AT, Reuser AJ.

J Inherit Metab Dis. 2015 Mar;38(2):305-14. doi: 10.1007/s10545-014-9707-6. Epub 2014 Apr 9.

18.

Synergistic Efficacy from Gene Therapy with Coreceptor Blockade and a β2-Agonist in Murine Pompe Disease.

Han SO, Li S, Bird A, Koeberl D.

Hum Gene Ther. 2015 Nov;26(11):743-50. doi: 10.1089/hum.2015.033. Epub 2015 Sep 29.

19.

Atypical immunologic response in a patient with CRIM-negative Pompe disease.

Abbott MA, Prater SN, Banugaria SG, Richards SM, Young SP, Rosenberg AS, Kishnani PS.

Mol Genet Metab. 2011 Dec;104(4):583-6. doi: 10.1016/j.ymgme.2011.08.003. Epub 2011 Aug 11.

20.

Persistence of high sustained antibodies to enzyme replacement therapy despite extensive immunomodulatory therapy in an infant with Pompe disease: need for agents to target antibody-secreting plasma cells.

Banugaria SG, Patel TT, Mackey J, Das S, Amalfitano A, Rosenberg AS, Charrow J, Chen YT, Kishnani PS.

Mol Genet Metab. 2012 Apr;105(4):677-80. doi: 10.1016/j.ymgme.2012.01.019. Epub 2012 Jan 28.

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