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Items: 1 to 20 of 86

1.

Immune Modulation Therapy in a CRIM-Positive and IgG Antibody-Positive Infant with Pompe Disease Treated with Alglucosidase Alfa: A Case Report.

Markic J, Polic B, Kuzmanic-Samija R, Marusic E, Stricevic L, Metlicic V, Mestrovic J.

JIMD Rep. 2012;2:11-5. doi: 10.1007/8904_2011_34. Epub 2011 Sep 6.

2.

Effects of immune modulation therapy in the first Croatian infant diagnosed with Pompe disease: a 3-year follow-up study.

Markic J, Polic B, Stricevic L, Metlicic V, Kuzmanic-Samija R, Kovacevic T, Ivkosic IE, Mestrovic J.

Wien Klin Wochenschr. 2014 Feb;126(3-4):133-7. doi: 10.1007/s00508-013-0475-3. Epub 2013 Dec 14.

PMID:
24337590
3.

Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk.

Van den Hout JM, Kamphoven JH, Winkel LP, Arts WF, De Klerk JB, Loonen MC, Vulto AG, Cromme-Dijkhuis A, Weisglas-Kuperus N, Hop W, Van Hirtum H, Van Diggelen OP, Boer M, Kroos MA, Van Doorn PA, Van der Voort E, Sibbles B, Van Corven EJ, Brakenhoff JP, Van Hove J, Smeitink JA, de Jong G, Reuser AJ, Van der Ploeg AT.

Pediatrics. 2004 May;113(5):e448-57.

PMID:
15121988
4.

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
5.

High dose IVIG successfully reduces high rhGAA IgG antibody titers in a CRIM-negative infantile Pompe disease patient.

Rairikar M, Kazi ZB, Desai A, Walters C, Rosenberg A, Kishnani PS.

Mol Genet Metab. 2017 May 18. pii: S1096-7192(17)30163-4. doi: 10.1016/j.ymgme.2017.05.006. [Epub ahead of print]

PMID:
28648664
6.

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.

7.

High antibody titer in an adult with Pompe disease affects treatment with alglucosidase alfa.

de Vries JM, van der Beek NA, Kroos MA, Ozkan L, van Doorn PA, Richards SM, Sung CC, Brugma JD, Zandbergen AA, van der Ploeg AT, Reuser AJ.

Mol Genet Metab. 2010 Dec;101(4):338-45. doi: 10.1016/j.ymgme.2010.08.009. Epub 2010 Aug 14.

PMID:
20826098
8.

Sustained immune tolerance induction in enzyme replacement therapy-treated CRIM-negative patients with infantile Pompe disease.

Kazi ZB, Desai AK, Berrier KL, Troxler RB, Wang RY, Abdul-Rahman OA, Tanpaiboon P, Mendelsohn NJ, Herskovitz E, Kronn D, Inbar-Feigenberg M, Ward-Melver C, Polan M, Gupta P, Rosenberg AS, Kishnani PS.

JCI Insight. 2017 Aug 17;2(16). pii: 94328. doi: 10.1172/jci.insight.94328. [Epub ahead of print]

PMID:
28814660
9.

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.

10.

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.

11.

Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease.

Sun B, Banugaria SG, Prater SN, Patel TT, Fredrickson K, Ringler DJ, de Fougerolles A, Rosenberg AS, Waldmann H, Kishnani PS.

Mol Genet Metab Rep. 2014 Oct 12;1:446-450. eCollection 2014.

12.

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.

13.

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.

14.

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.

15.

Alglucosidase alfa: new drug. Pompe disease: a short-term benefit.

[No authors listed]

Prescrire Int. 2007 Dec;16(92):240-1.

PMID:
18092404
16.

Successful Desensitisation in a Patient with CRIM-Positive Infantile-Onset Pompe Disease.

Baruteau J, Broomfield A, Crook V, Finnegan N, Harvey K, Burke D, Burch M, Shepherd G, Vellodi A.

JIMD Rep. 2014;12:99-102. doi: 10.1007/8904_2013_250. Epub 2013 Sep 4.

17.
18.

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
19.

Algorithm for the early diagnosis and treatment of patients with cross reactive immunologic material-negative classic infantile pompe disease: a step towards improving the efficacy of ERT.

Banugaria SG, Prater SN, Patel TT, Dearmey SM, Milleson C, Sheets KB, Bali DS, Rehder CW, Raiman JA, Wang RA, Labarthe F, Charrow J, Harmatz P, Chakraborty P, Rosenberg AS, Kishnani PS.

PLoS One. 2013 Jun 25;8(6):e67052. doi: 10.1371/journal.pone.0067052. Print 2013.

20.

Bortezomib in the rapid reduction of high sustained antibody titers in disorders treated with therapeutic protein: lessons learned from Pompe disease.

Banugaria SG, Prater SN, McGann JK, Feldman JD, Tannenbaum JA, Bailey C, Gera R, Conway RL, Viskochil D, Kobori JA, Rosenberg AS, Kishnani PS.

Genet Med. 2013 Feb;15(2):123-31. doi: 10.1038/gim.2012.110. Epub 2012 Oct 11.

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