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Items: 1 to 20 of 279

1.

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
2.

Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle.

Koeberl DD, Luo X, Sun B, McVie-Wylie A, Dai J, Li S, Banugaria SG, Chen YT, Bali DS.

Mol Genet Metab. 2011 Jun;103(2):107-12. doi: 10.1016/j.ymgme.2011.02.006. Epub 2011 Feb 13.

3.

A beta-blocker, propranolol, decreases the efficacy from enzyme replacement therapy in Pompe disease.

Han SO, Pope R, Li S, Kishnani PS, Steet R, Koeberl DD.

Mol Genet Metab. 2016 Feb;117(2):114-9. doi: 10.1016/j.ymgme.2015.09.012. Epub 2015 Oct 3.

4.

New therapeutic approaches for Pompe disease: enzyme replacement therapy and beyond.

Kishnani PS, Beckemeyer AA.

Pediatr Endocrinol Rev. 2014 Sep;12 Suppl 1:114-24. Review.

PMID:
25345093
5.

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
6.

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
7.

Effects of exercise training during infusion on late-onset Pompe disease patients receiving enzyme replacement therapy.

Terzis G, Krase A, Papadimas G, Papadopoulos C, Kavouras SA, Manta P.

Mol Genet Metab. 2012 Dec;107(4):669-73. doi: 10.1016/j.ymgme.2012.10.020. Epub 2012 Oct 24.

PMID:
23146291
8.

Enzyme replacement therapy for Pompe disease.

Angelini C, Semplicini C.

Curr Neurol Neurosci Rep. 2012 Feb;12(1):70-5. doi: 10.1007/s11910-011-0236-5. Review.

PMID:
22002767
9.

Combined aerobic exercise and enzyme replacement therapy rejuvenates the mitochondrial-lysosomal axis and alleviates autophagic blockage in Pompe disease.

Nilsson MI, MacNeil LG, Kitaoka Y, Suri R, Young SP, Kaczor JJ, Nates NJ, Ansari MU, Wong T, Ahktar M, Brandt L, Hettinga BP, Tarnopolsky MA.

Free Radic Biol Med. 2015 Oct;87:98-112. doi: 10.1016/j.freeradbiomed.2015.05.019. Epub 2015 May 19.

PMID:
26001726
10.

Infantile Pompe disease on ERT: update on clinical presentation, musculoskeletal management, and exercise considerations.

Case LE, Beckemeyer AA, Kishnani PS.

Am J Med Genet C Semin Med Genet. 2012 Feb 15;160C(1):69-79. doi: 10.1002/ajmg.c.31321. Epub 2012 Jan 17. Review.

PMID:
22252989
11.

Effect of aerobic and resistance exercise training on late-onset Pompe disease patients receiving enzyme replacement therapy.

Terzis G, Dimopoulos F, Papadimas GK, Papadopoulos C, Spengos K, Fatouros I, Kavouras SA, Manta P.

Mol Genet Metab. 2011 Nov;104(3):279-83. doi: 10.1016/j.ymgme.2011.05.013. Epub 2011 May 19.

PMID:
21640624
12.

β2 Agonists enhance the efficacy of simultaneous enzyme replacement therapy in murine Pompe disease.

Koeberl DD, Li S, Dai J, Thurberg BL, Bali D, Kishnani PS.

Mol Genet Metab. 2012 Feb;105(2):221-7. doi: 10.1016/j.ymgme.2011.11.005. Epub 2011 Nov 11.

13.

Inhibition of glycogen biosynthesis via mTORC1 suppression as an adjunct therapy for Pompe disease.

Ashe KM, Taylor KM, Chu Q, Meyers E, Ellis A, Jingozyan V, Klinger K, Finn PF, Cooper CG, Chuang WL, Marshall J, McPherson JM, Mattaliano RJ, Cheng SH, Scheule RK, Moreland RJ.

Mol Genet Metab. 2010 Aug;100(4):309-15. doi: 10.1016/j.ymgme.2010.05.001. Epub 2010 May 5.

PMID:
20554235
14.

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
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.

Quantitative computed tomography for enzyme replacement therapy in Pompe disease.

Yonee C, Toyoshima M, Young SP, Maruyama S, Higuchi I, Narita A, Maegaki Y, Nanba E, Ohno K, Kawano Y.

Brain Dev. 2012 Nov;34(10):834-9. doi: 10.1016/j.braindev.2012.01.013. Epub 2012 Apr 21.

PMID:
22521436
17.

Enzyme replacement therapy in severe adult-onset glycogen storage disease type II.

Ravaglia S, Danesino C, Pichiecchio A, Repetto A, Poloni GU, Rossi M, Fratino P, Moglia A, Costa A.

Adv Ther. 2008 Aug;25(8):820-9. doi: 10.1007/s12325-008-0086-y.

PMID:
18704279
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.

Enzyme replacement therapy and fatigue in adults with Pompe disease.

Güngör D, de Vries JM, Brusse E, Kruijshaar ME, Hop WC, Murawska M, van den Berg LE, Reuser AJ, van Doorn PA, Hagemans ML, Plug I, van der Ploeg AT.

Mol Genet Metab. 2013 Jun;109(2):174-8. doi: 10.1016/j.ymgme.2013.03.016. Epub 2013 Apr 3.

PMID:
23603069
20.

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

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