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Results: 1 to 20 of 210

1.

Both type 1 and type 2a muscle fibers can respond to enzyme therapy in Pompe disease.

Drost MR, Schaart G, van Dijk P, van Capelle CI, van der Vusse GJ, Delhaas T, van der Ploeg AT, Reuser AJ.

Muscle Nerve. 2008 Feb;37(2):251-5.

PMID:
17894362
[PubMed - indexed for MEDLINE]
2.

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.

Lab Invest. 2006 Dec;86(12):1208-20. Epub 2006 Oct 30.

PMID:
17075580
[PubMed - indexed for MEDLINE]
Free Article
3.

Replacing acid alpha-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers.

Raben N, Fukuda T, Gilbert AL, de Jong D, Thurberg BL, Mattaliano RJ, Meikle P, Hopwood JJ, Nagashima K, Nagaraju K, Plotz PH.

Mol Ther. 2005 Jan;11(1):48-56.

PMID:
15585405
[PubMed - indexed for MEDLINE]
4.

Murine muscle cell models for Pompe disease and their use in studying therapeutic approaches.

Takikita S, Myerowitz R, Zaal K, Raben N, Plotz PH.

Mol Genet Metab. 2009 Apr;96(4):208-17. doi: 10.1016/j.ymgme.2008.12.012. Epub 2009 Jan 22.

PMID:
19167256
[PubMed - indexed for MEDLINE]
Free PMC Article
5.

Suppression of autophagy permits successful enzyme replacement therapy in a lysosomal storage disorder--murine Pompe disease.

Raben N, Schreiner C, Baum R, Takikita S, Xu S, Xie T, Myerowitz R, Komatsu M, Van der Meulen JH, Nagaraju K, Ralston E, Plotz PH.

Autophagy. 2010 Nov;6(8):1078-89. doi: 10.4161/auto.6.8.13378.

PMID:
20861693
[PubMed - indexed for MEDLINE]
Free PMC Article
6.

A case of adult Pompe disease presenting with severe fatigue and selective involvement of type 1 muscle fibers.

van den Berg LE, de Vries JM, Verdijk RM, van der Ploeg AT, Reuser AJ, van Doorn PA.

Neuromuscul Disord. 2011 Mar;21(3):232-4. doi: 10.1016/j.nmd.2010.11.016. Epub 2010 Dec 22.

PMID:
21183346
[PubMed - indexed for MEDLINE]
Free Article
7.

Autophagy and lysosomes in Pompe disease.

Fukuda T, Roberts A, Ahearn M, Zaal K, Ralston E, Plotz PH, Raben N.

Autophagy. 2006 Oct-Dec;2(4):318-20. Epub 2006 Oct 5.

PMID:
16874053
[PubMed - indexed for MEDLINE]
Free Article
8.

Aerobic training as an adjunctive therapy to enzyme replacement in Pompe disease.

Nilsson MI, Samjoo IA, Hettinga BP, Koeberl DD, Zhang H, Hawke TJ, Nissar AA, Ali T, Brandt L, Ansari MU, Hazari H, Patel N, Amon J, Tarnopolsky MA.

Mol Genet Metab. 2012 Nov;107(3):469-79. doi: 10.1016/j.ymgme.2012.09.010. Epub 2012 Sep 15.

PMID:
23041258
[PubMed - indexed for MEDLINE]
9.

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

Hawes ML, Kennedy W, O'Callaghan MW, Thurberg BL.

Mol Genet Metab. 2007 Aug;91(4):343-51. Epub 2007 Jun 14.

PMID:
17572127
[PubMed - indexed for MEDLINE]
10.

[Clinical development of acid alpha-glucosidase for the treatment of Pompe disease].

Nicolino M.

Presse Med. 2007 Mar;36 Spec No 1:1S100-7. Review. French.

PMID:
17546777
[PubMed - indexed for MEDLINE]
11.

Morphological changes in muscle tissue of patients with infantile Pompe's disease receiving enzyme replacement therapy.

Winkel LP, Kamphoven JH, van den Hout HJ, Severijnen LA, van Doorn PA, Reuser AJ, van der Ploeg AT.

Muscle Nerve. 2003 Jun;27(6):743-51.

PMID:
12766987
[PubMed - indexed for MEDLINE]
12.

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
[PubMed - indexed for MEDLINE]
13.

Improved efficacy of gene therapy approaches for Pompe disease using a new, immune-deficient GSD-II mouse model.

Xu F, Ding E, Liao SX, Migone F, Dai J, Schneider A, Serra D, Chen YT, Amalfitano A.

Gene Ther. 2004 Nov;11(21):1590-8.

PMID:
15356673
[PubMed - indexed for MEDLINE]
14.

[Enzyme replacement therapy in a patient with Pompe disease].

Fujikawa Y, Kinoshita S, Miyamoto Y, Nakayama T, Endo Y, Sasaki M.

No To Hattatsu. 2007 Sep;39(5):383-6. Japanese.

PMID:
17879614
[PubMed - indexed for MEDLINE]
15.

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.

Mol Genet Metab. 2003 Sep-Oct;80(1-2):159-69.

PMID:
14567965
[PubMed - indexed for MEDLINE]
16.

24-months results in two adults with Pompe disease on enzyme replacement therapy.

Vielhaber S, Brejova A, Debska-Vielhaber G, Kaufmann J, Feistner H, Schoenfeld MA, Awiszus F.

Clin Neurol Neurosurg. 2011 Jun;113(5):350-7. doi: 10.1016/j.clineuro.2010.09.016. Epub 2011 Apr 7.

PMID:
21477922
[PubMed - indexed for MEDLINE]
17.

Deconstructing Pompe disease by analyzing single muscle fibers: to see a world in a grain of sand...

Raben N, Takikita S, Pittis MG, Bembi B, Marie SK, Roberts A, Page L, Kishnani PS, Schoser BG, Chien YH, Ralston E, Nagaraju K, Plotz PH.

Autophagy. 2007 Nov-Dec;3(6):546-52. Epub 2007 Jun 15.

PMID:
17592248
[PubMed - indexed for MEDLINE]
Free Article
18.

Enzyme therapy for pompe disease with recombinant human alpha-glucosidase from rabbit milk.

Van den Hout JM, Reuser AJ, de Klerk JB, Arts WF, Smeitink JA, Van der Ploeg AT.

J Inherit Metab Dis. 2001 Apr;24(2):266-74.

PMID:
11405345
[PubMed - indexed for MEDLINE]
19.

Prognostic factors for the late onset Pompe disease with enzyme replacement therapy: from our experience of 4 cases including an autopsy case.

Kobayashi H, Shimada Y, Ikegami M, Kawai T, Sakurai K, Urashima T, Ijima M, Fujiwara M, Kaneshiro E, Ohashi T, Eto Y, Ishigaki K, Osawa M, Kyosen SO, Ida H.

Mol Genet Metab. 2010 May;100(1):14-9. doi: 10.1016/j.ymgme.2010.01.015. Epub 2010 Feb 4.

PMID:
20202878
[PubMed - indexed for MEDLINE]
20.

Autophagy in skeletal muscle: implications for Pompe disease.

Shea L, Raben N.

Int J Clin Pharmacol Ther. 2009;47 Suppl 1:S42-7. Review.

PMID:
20040311
[PubMed - indexed for MEDLINE]
Free PMC Article

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