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Items: 1 to 20 of 107

1.

First experience with enzyme replacement therapy during pregnancy and lactation in Pompe disease.

de Vries JM, Brugma JD, Ozkan L, Steegers EA, Reuser AJ, van Doorn PA, van der Ploeg AT.

Mol Genet Metab. 2011 Dec;104(4):552-5. doi: 10.1016/j.ymgme.2011.09.012. Epub 2011 Sep 16.

PMID:
21967859
2.

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
3.

Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease.

Case LE, Bjartmar C, Morgan C, Casey R, Charrow J, Clancy JP, Dasouki M, DeArmey S, Nedd K, Nevins M, Peters H, Phillips D, Spigelman Z, Tifft C, Kishnani PS.

Neuromuscul Disord. 2015 Apr;25(4):321-32. doi: 10.1016/j.nmd.2014.12.004. Epub 2014 Dec 19.

4.

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

[No authors listed]

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

PMID:
18092404
5.

Desensitization of an adult patient with Pompe disease and a history of anaphylaxis to alglucosidase alfa.

Lipinski SE, Lipinski MJ, Burnette A, Platts-Mills TA, Wilson WG.

Mol Genet Metab. 2009 Nov;98(3):319-21. doi: 10.1016/j.ymgme.2009.07.001. Epub 2009 Jul 7.

PMID:
19640753
6.

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
7.

36 months observational clinical study of 38 adult Pompe disease patients under alglucosidase alfa enzyme replacement therapy.

Regnery C, Kornblum C, Hanisch F, Vielhaber S, Strigl-Pill N, Grunert B, Müller-Felber W, Glocker FX, Spranger M, Deschauer M, Mengel E, Schoser B.

J Inherit Metab Dis. 2012 Sep;35(5):837-45. Epub 2012 Jan 31.

PMID:
22290025
8.

Early administration of enzyme replacement therapy for Pompe disease: short-term follow-up results.

Hamdan MA, Almalik MH, Mirghani HM.

J Inherit Metab Dis. 2008 Dec;31 Suppl 2:S431-6. doi: 10.1007/s10545-008-1000-0. Epub 2008 Dec 12.

PMID:
19067231
9.

Sudden deterioration in nonclassical infantile-onset Pompe disease responding to alglucosidase alfa infusion therapy: a case report.

Pascual-Pascual SI, Rubio P, Albajara L, Gutierrez M, Chabas A, Alvarado F.

J Inherit Metab Dis. 2006 Dec;29(6):763. Epub 2006 Oct 14.

PMID:
17041744
10.

An individually, modified approach to desensitize infants and young children with Pompe disease, and significant reactions to alglucosidase alfa infusions.

El-Gharbawy AH, Mackey J, DeArmey S, Westby G, Grinnell SG, Malovrh P, Conway R, Kishnani PS.

Mol Genet Metab. 2011 Sep-Oct;104(1-2):118-22. doi: 10.1016/j.ymgme.2011.07.004. Epub 2011 Jul 13.

11.
12.

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
13.

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
14.

Early cognitive development in children with infantile Pompe disease.

Spiridigliozzi GA, Heller JH, Case LE, Jones HN, Kishnani PS.

Mol Genet Metab. 2012 Mar;105(3):428-32. doi: 10.1016/j.ymgme.2011.10.012. Epub 2011 Oct 28.

PMID:
22217428
15.

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
16.

Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease.

Kanters TA, Hoogenboom-Plug I, Rutten-Van Mölken MP, Redekop WK, van der Ploeg AT, Hakkaart L.

Orphanet J Rare Dis. 2014 May 16;9:75. doi: 10.1186/1750-1172-9-75.

17.

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.

18.

Pregnancy during enzyme replacement therapy for late-onset acid maltase deficiency.

Zagnoli F, Leblanc A, Blanchard C.

Neuromuscul Disord. 2013 Feb;23(2):180-1. doi: 10.1016/j.nmd.2012.11.006. Epub 2013 Jan 3.

PMID:
23290485
19.

Alglucosidase alfa and Pompe disease: still going strong?

Lipinski SE.

Mol Genet Metab. 2012 Nov;107(3):245-6. doi: 10.1016/j.ymgme.2012.09.014. Epub 2012 Sep 17. No abstract available.

PMID:
23034445
20.

Cessation and resuming of alglucosidase alfa in Pompe disease: a retrospective analysis.

Hundsberger T, Rösler KM, Findling O.

J Neurol. 2014 Sep;261(9):1684-90. doi: 10.1007/s00415-014-7402-z. Epub 2014 Jun 13.

PMID:
24923245

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