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Results: 1 to 20 of 106

1.

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
[PubMed - indexed for MEDLINE]
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
[PubMed - indexed for MEDLINE]
3.

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.

PMID:
20393176
[PubMed - indexed for MEDLINE]
Free Article
4.

Early treatment with alglucosidase alpha prolongs long-term survival of infants with Pompe disease.

Kishnani PS, Corzo D, Leslie ND, Gruskin D, Van der Ploeg A, Clancy JP, Parini R, Morin G, Beck M, Bauer MS, Jokic M, Tsai CE, Tsai BW, Morgan C, O'Meara T, Richards S, Tsao EC, Mandel H.

Pediatr Res. 2009 Sep;66(3):329-35. doi: 10.1203/PDR.0b013e3181b24e94.

PMID:
19542901
[PubMed - indexed for MEDLINE]
Free PMC Article
5.

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.

PMID:
21802969
[PubMed - indexed for MEDLINE]
Free PMC Article
6.

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

[No authors listed]

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

PMID:
18092404
[PubMed - indexed for MEDLINE]
7.

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.

PMID:
16860134
[PubMed - indexed for MEDLINE]
Free PMC Article
8.

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
[PubMed - indexed for MEDLINE]
9.

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
[PubMed - indexed for MEDLINE]
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
[PubMed - indexed for MEDLINE]
11.

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
[PubMed - indexed for MEDLINE]
12.

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
[PubMed - indexed for MEDLINE]
13.

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
[PubMed - indexed for MEDLINE]
14.

Observational clinical study in juvenile-adult glycogenosis type 2 patients undergoing enzyme replacement therapy for up to 4 years.

Angelini C, Semplicini C, Ravaglia S, Bembi B, Servidei S, Pegoraro E, Moggio M, Filosto M, Sette E, Crescimanno G, Tonin P, Parini R, Morandi L, Marrosu G, Greco G, Musumeci O, Di Iorio G, Siciliano G, Donati MA, Carubbi F, Ermani M, Mongini T, Toscano A; Italian GSDII Group.

J Neurol. 2012 May;259(5):952-8. doi: 10.1007/s00415-011-6293-5. Epub 2011 Nov 12.

PMID:
22081099
[PubMed - indexed for MEDLINE]
15.

B-Cell depletion and immunomodulation before initiation of enzyme replacement therapy blocks the immune response to acid alpha-glucosidase in infantile-onset Pompe disease.

Elder ME, Nayak S, Collins SW, Lawson LA, Kelley JS, Herzog RW, Modica RF, Lew J, Lawrence RM, Byrne BJ.

J Pediatr. 2013 Sep;163(3):847-54.e1. doi: 10.1016/j.jpeds.2013.03.002. Epub 2013 Apr 16.

PMID:
23601496
[PubMed - indexed for MEDLINE]
Free PMC Article
16.

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
[PubMed - indexed for MEDLINE]
17.

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.

PMID:
18661169
[PubMed - indexed for MEDLINE]
Free PMC Article
18.

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
[PubMed - indexed for MEDLINE]
19.

Long-term follow-up results in enzyme replacement therapy for Pompe disease: a case report.

Del Rizzo M, Fanin M, Cerutti A, Cazzorla C, Milanesi O, Nascimbeni AC, Angelini C, Giordano L, Bordugo A, Burlina AB.

J Inherit Metab Dis. 2010 Dec;33 Suppl 3:S389-93. doi: 10.1007/s10545-010-9195-2. Epub 2010 Sep 10.

PMID:
20830524
[PubMed - indexed for MEDLINE]
20.

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
[PubMed - indexed for MEDLINE]

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