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Items: 1 to 20 of 84

1.

Desensitisation of the youngest patient with Pompe disease in response to alglucosidase alfa.

Karagol IH, Bakirtas A, Yilmaz O, Topal E, Kucukcongar A, Ezgu FS, Demirsoy MS, Turktas I.

Allergol Immunopathol (Madr). 2014 Jul-Aug;42(4):372-5. doi: 10.1016/j.aller.2013.02.012. Epub 2013 Jun 14. No abstract available.

PMID:
23769736
2.

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
3.

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

[No authors listed]

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

PMID:
18092404
4.

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.

5.

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
6.

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
7.

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
8.

A randomized study of alglucosidase alfa in late-onset Pompe's disease.

van der Ploeg AT, Clemens PR, Corzo D, Escolar DM, Florence J, Groeneveld GJ, Herson S, Kishnani PS, Laforet P, Lake SL, Lange DJ, Leshner RT, Mayhew JE, Morgan C, Nozaki K, Park DJ, Pestronk A, Rosenbloom B, Skrinar A, van Capelle CI, van der Beek NA, Wasserstein M, Zivkovic SA.

N Engl J Med. 2010 Apr 15;362(15):1396-406. doi: 10.1056/NEJMoa0909859.

9.

Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease.

Nicolino M, Byrne B, Wraith JE, Leslie N, Mandel H, Freyer DR, Arnold GL, Pivnick EK, Ottinger CJ, Robinson PH, Loo JC, Smitka M, Jardine P, Tatò L, Chabrol B, McCandless S, Kimura S, Mehta L, Bali D, Skrinar A, Morgan C, Rangachari L, Corzo D, Kishnani PS.

Genet Med. 2009 Mar;11(3):210-9. doi: 10.1097/GIM.0b013e31819d0996.

PMID:
19287243
10.

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
11.

Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa.

van der Ploeg AT, Barohn R, Carlson L, Charrow J, Clemens PR, Hopkin RJ, Kishnani PS, Laforêt P, Morgan C, Nations S, Pestronk A, Plotkin H, Rosenbloom BE, Sims KB, Tsao E.

Mol Genet Metab. 2012 Nov;107(3):456-61. doi: 10.1016/j.ymgme.2012.09.015. Epub 2012 Sep 17.

PMID:
23031366
12.

First treatment for Pompe disease.

[No authors listed]

FDA Consum. 2006 Jul-Aug;40(4):3. No abstract available.

13.

alpha-glucosidase (CHO) (Genzyme).

Lachmann RH.

Curr Opin Investig Drugs. 2004 Oct;5(10):1101-10. Review.

PMID:
15535432
14.

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
15.

Anaesthetic management of infants with glycogen storage disease type II: a physiological approach.

Ing RJ, Cook DR, Bengur RA, Williams EA, Eck J, Dear Gde L, Ross AK, Kern FH, Kishnani PS.

Paediatr Anaesth. 2004 Jun;14(6):514-9.

PMID:
15153218
16.

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.

17.

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
18.

Enzyme replacement in Pompe disease with an alpha-glucosidase-low density lipoprotein complex.

Williams JC, Murray AK.

Birth Defects Orig Artic Ser. 1980;16(1):415-23. No abstract available.

PMID:
7004520
19.

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
20.

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

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