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Items: 1 to 20 of 116

1.

Immune tolerance induction to enzyme-replacement therapy by co-administration of short-term, low-dose methotrexate in a murine Pompe disease model.

Joseph A, Munroe K, Housman M, Garman R, Richards S.

Clin Exp Immunol. 2008 Apr;152(1):138-46. doi: 10.1111/j.1365-2249.2008.03602.x. Epub 2008 Feb 25.

2.

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
3.

Nephrotic syndrome complicating alpha-glucosidase replacement therapy for Pompe disease.

Hunley TE, Corzo D, Dudek M, Kishnani P, Amalfitano A, Chen YT, Richards SM, Phillips JA 3rd, Fogo AB, Tiller GE.

Pediatrics. 2004 Oct;114(4):e532-5.

PMID:
15466083
4.

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.

5.

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.

6.

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.

7.

Transient low-dose methotrexate generates B regulatory cells that mediate antigen-specific tolerance to alglucosidase alfa.

Joly MS, Martin RP, Mitra-Kaushik S, Phillips L, D'Angona A, Richards SM, Joseph AM.

J Immunol. 2014 Oct 15;193(8):3947-58. doi: 10.4049/jimmunol.1303326. Epub 2014 Sep 10.

8.

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.

9.

Oral administration of recombinant human acid α-glucosidase reduces specific antibody formation against enzyme in mouse.

Ohashi T, Iizuka S, Shimada Y, Eto Y, Ida H, Hachimura S, Kobayashi H.

Mol Genet Metab. 2011 May;103(1):98-100. doi: 10.1016/j.ymgme.2011.01.009. Epub 2011 Jan 27.

PMID:
21320791
10.

Hyaluronidase increases the biodistribution of acid alpha-1,4 glucosidase in the muscle of Pompe disease mice: an approach to enhance the efficacy of enzyme replacement therapy.

Matalon R, Surendran S, Campbell GA, Michals-Matalon K, Tyring SK, Grady J, Cheng S, Kaye E.

Biochem Biophys Res Commun. 2006 Nov 24;350(3):783-7. Epub 2006 Oct 2.

PMID:
17027913
11.

Safety and efficacy of recombinant acid alpha-glucosidase (rhGAA) in patients with classical infantile Pompe disease: results of a phase II clinical trial.

Klinge L, Straub V, Neudorf U, Schaper J, Bosbach T, Görlinger K, Wallot M, Richards S, Voit T.

Neuromuscul Disord. 2005 Jan;15(1):24-31. Epub 2004 Nov 26.

PMID:
15639117
12.

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.

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.

Recombinant human acid alpha-glucosidase corrects acid alpha-glucosidase-deficient human fibroblasts, quail fibroblasts, and quail myoblasts.

Yang HW, Kikuchi T, Hagiwara Y, Mizutani M, Chen YT, Van Hove JL.

Pediatr Res. 1998 Mar;43(3):374-80.

PMID:
9505277
15.

Immune tolerance induced using plasma exchange and rituximab in an infantile Pompe disease patient.

Deodato F, Ginocchio VM, Onofri A, Grutter G, Germani A, Dionisi-Vici C.

J Child Neurol. 2014 Jun;29(6):850-4. doi: 10.1177/0883073813485819. Epub 2013 Apr 25.

PMID:
23620524
16.

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.

17.

Long-term enzyme replacement therapy for pompe disease with recombinant human alpha-glucosidase derived from chinese hamster ovary cells.

Rossi M, Parenti G, Della Casa R, Romano A, Mansi G, Agovino T, Rosapepe F, Vosa C, Del Giudice E, Andria G.

J Child Neurol. 2007 May;22(5):565-73.

PMID:
17690063
18.

Human acid alpha-glucosidase from rabbit milk has therapeutic effect in mice with glycogen storage disease type II.

Bijvoet AG, Van Hirtum H, Kroos MA, Van de Kamp EH, Schoneveld O, Visser P, Brakenhoff JP, Weggeman M, van Corven EJ, Van der Ploeg AT, Reuser AJ.

Hum Mol Genet. 1999 Nov;8(12):2145-53.

PMID:
10545593
19.

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.

20.

Report of the first Brazilian infantile Pompe disease patient to be treated with recombinant human acid alpha-glucosidase.

Pereira SJ, Berditchevisky CR, Marie SK.

J Pediatr (Rio J). 2008 May-Jun;84(3):272-5. doi: doi:10.2223/JPED.1793.

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