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Items: 1 to 20 of 283

1.

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.

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.

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
4.

Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial.

Amalfitano A, Bengur AR, Morse RP, Majure JM, Case LE, Veerling DL, Mackey J, Kishnani P, Smith W, McVie-Wylie A, Sullivan JA, Hoganson GE, Phillips JA 3rd, Schaefer GB, Charrow J, Ware RE, Bossen EH, Chen YT.

Genet Med. 2001 Mar-Apr;3(2):132-8.

PMID:
11286229
5.

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
6.

Cardiac remodeling after enzyme replacement therapy with acid alpha-glucosidase for infants with Pompe disease.

Levine JC, Kishnani PS, Chen YT, Herlong JR, Li JS.

Pediatr Cardiol. 2008 Nov;29(6):1033-42. doi: 10.1007/s00246-008-9267-3. Epub 2008 Jul 26.

7.

[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
8.

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
9.

Long-term enzyme replacement therapy for pompe disease with recombinant human alpha-glucosidase derived from chinese hamster ovary cells.

Rossi M, Parenti G, Della Casa R, Romano A, Mansi G, Agovino T, Rosapepe F, Vosa C, Del Giudice E, Andria G.

J Child Neurol. 2007 May;22(5):565-73.

PMID:
17690063
10.

Electrocardiographic response to enzyme replacement therapy for Pompe disease.

Ansong AK, Li JS, Nozik-Grayck E, Ing R, Kravitz RM, Idriss SF, Kanter RJ, Rice H, Chen YT, Kishnani PS.

Genet Med. 2006 May;8(5):297-301.

PMID:
16702879
11.

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
12.

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
13.

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
14.

CRIM-negative infantile Pompe disease: 42-month treatment outcome.

Rohrbach M, Klein A, Köhli-Wiesner A, Veraguth D, Scheer I, Balmer C, Lauener R, Baumgartner MR.

J Inherit Metab Dis. 2010 Dec;33(6):751-7. doi: 10.1007/s10545-010-9209-0. Epub 2010 Sep 30.

PMID:
20882352
15.

A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease.

Kishnani PS, Hwu WL, Mandel H, Nicolino M, Yong F, Corzo D; Infantile-Onset Pompe Disease Natural History Study Group.

J Pediatr. 2006 May;148(5):671-676.

PMID:
16737883
16.

Fractures in children with Pompe disease: a potential long-term complication.

Case LE, Hanna R, Frush DP, Krishnamurthy V, DeArmey S, Mackey J, Boney A, Morgan C, Corzo D, Bouchard S, Weber TJ, Chen YT, Kishnani PS.

Pediatr Radiol. 2007 May;37(5):437-45. Epub 2007 Mar 7.

PMID:
17342521
17.

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.

18.

Treatment of infantile Pompe disease with alglucosidase alpha: the UK experience.

Chakrapani A, Vellodi A, Robinson P, Jones S, Wraith JE.

J Inherit Metab Dis. 2010 Dec;33(6):747-50. doi: 10.1007/s10545-010-9206-3. Epub 2010 Sep 24.

PMID:
20865334
19.

Report of the first Brazilian infantile Pompe disease patient to be treated with recombinant human acid alpha-glucosidase.

Pereira SJ, Berditchevisky CR, Marie SK.

J Pediatr (Rio J). 2008 May-Jun;84(3):272-5. doi: doi:10.2223/JPED.1793.

20.

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
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