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Items: 1 to 20 of 435

1.

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.

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.

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
4.

High-resolution light microscopy (HRLM) and digital analysis of Pompe disease pathology.

Lynch CM, Johnson J, Vaccaro C, Thurberg BL.

J Histochem Cytochem. 2005 Jan;53(1):63-73.

PMID:
15637339
5.

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
6.

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
7.

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
8.

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
9.

Pompe disease (glycogen storage disease type II): clinical features and enzyme replacement therapy.

van der Beek NA, Hagemans ML, van der Ploeg AT, Reuser AJ, van Doorn PA.

Acta Neurol Belg. 2006 Jun;106(2):82-6. Review.

PMID:
16898258
10.

Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease.

Kishnani PS, Corzo D, Nicolino M, Byrne B, Mandel H, Hwu WL, Leslie N, Levine J, Spencer C, McDonald M, Li J, Dumontier J, Halberthal M, Chien YH, Hopkin R, Vijayaraghavan S, Gruskin D, Bartholomew D, van der Ploeg A, Clancy JP, Parini R, Morin G, Beck M, De la Gastine GS, Jokic M, Thurberg B, Richards S, Bali D, Davison M, Worden MA, Chen YT, Wraith JE.

Neurology. 2007 Jan 9;68(2):99-109. Epub 2006 Dec 6. Erratum in: Neurology. 2008 Nov 18;71(21):1748.

PMID:
17151339
11.

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
12.

Enzyme replacement therapy in classical infantile pompe disease: results of a ten-month follow-up study.

Klinge L, Straub V, Neudorf U, Voit T.

Neuropediatrics. 2005 Feb;36(1):6-11.

PMID:
15776317
13.

Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease.

Kishnani PS, Nicolino M, Voit T, Rogers RC, Tsai AC, Waterson J, Herman GE, Amalfitano A, Thurberg BL, Richards S, Davison M, Corzo D, Chen YT.

J Pediatr. 2006 Jul;149(1):89-97.

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
15.

[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
16.

Cardiac arrhythmias following anesthesia induction in infantile-onset Pompe disease: a case series.

Wang LY, Ross AK, Li JS, Dearmey SM, Mackey JF, Worden M, Corzo D, Morgan C, Kishnani PS.

Paediatr Anaesth. 2007 Aug;17(8):738-48.

PMID:
17596219
17.

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
18.

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.

19.

Enzyme replacement therapy with alglucosidase alfa in 44 patients with late-onset glycogen storage disease type 2: 12-month results of an observational clinical trial.

Strothotte S, Strigl-Pill N, Grunert B, Kornblum C, Eger K, Wessig C, Deschauer M, Breunig F, Glocker FX, Vielhaber S, Brejova A, Hilz M, Reiners K, Müller-Felber W, Mengel E, Spranger M, Schoser B.

J Neurol. 2010 Jan;257(1):91-7. doi: 10.1007/s00415-009-5275-3. Epub 2009 Aug 1.

PMID:
19649685
20.

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
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