These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.


BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

178 related articles for article (PubMed ID: 30778879)

  • 1. Desensitization of two young patients with infantile-onset Pompe disease and severe reactions to alglucosidase alfa.
    Gragnaniello V; Fecarotta S; Pecoraro A; Tarallo A; Catzola A; Spadaro G; Parenti G; Della Casa R
    Neurol Sci; 2019 Jul; 40(7):1453-1455. PubMed ID: 30778879
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Enzyme replacement therapy for infantile-onset Pompe disease.
    Chen M; Zhang L; Quan S
    Cochrane Database Syst Rev; 2017 Nov; 11(11):CD011539. PubMed ID: 29155436
    [TBL] [Abstract][Full Text] [Related]  

  • 3. 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; 104(1-2):118-22. PubMed ID: 21802969
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Alglucosidase alfa: new drug. Pompe disease: a short-term benefit.
    Prescrire Int; 2007 Dec; 16(92):240-1. PubMed ID: 18092404
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Enzyme replacement therapy with alglucosidase alfa in Pompe disease: Clinical experience with rate escalation.
    Desai AK; Walters CK; Cope HL; Kazi ZB; DeArmey SM; Kishnani PS
    Mol Genet Metab; 2018 Feb; 123(2):92-96. PubMed ID: 29289479
    [TBL] [Abstract][Full Text] [Related]  

  • 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. PubMed ID: 20826098
    [TBL] [Abstract][Full Text] [Related]  

  • 7. First successful concomitant therapy of immune tolerance induction therapy and desensitization in a CRIM-negative infantile Pompe patient.
    Emecen Sanli M; Ertoy Karagol HI; Kilic A; Aktasoglu E; Inci A; Okur I; Ezgu F; Tumer L
    J Pediatr Endocrinol Metab; 2022 Feb; 35(2):273-277. PubMed ID: 34561975
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Hypersensitivity infusion-associated reactions induced by enzyme replacement therapy in a cohort of patients with late-onset Pompe disease: An experience from the French Pompe Registry.
    Lessard LER; Tard C; Salort-Campana E; Sacconi S; Béhin A; Bassez G; Orlikowski D; Merle P; Nollet S; Gallay L; Bérard F; Robinson P; Bouhour F; Laforêt P
    Mol Genet Metab; 2023 Jul; 139(3):107611. PubMed ID: 37285781
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Acid alpha-glucosidase deficiency (Pompe disease).
    Fukuda T; Roberts A; Plotz PH; Raben N
    Curr Neurol Neurosci Rep; 2007 Jan; 7(1):71-7. PubMed ID: 17217857
    [TBL] [Abstract][Full Text] [Related]  

  • 10. 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. PubMed ID: 19640753
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study.
    Hahn SH; Kronn D; Leslie ND; Pena LDM; Tanpaiboon P; Gambello MJ; Gibson JB; Hillman R; Stockton DW; Day JW; Wang RY; An Haack K; Shafi R; Sparks S; Zhao Y; Wilson C; Kishnani PS;
    Genet Med; 2018 Oct; 20(10):1284-1294. PubMed ID: 29565424
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Improvement with ongoing Enzyme Replacement Therapy in advanced late-onset Pompe disease: a case study.
    Case LE; Koeberl DD; Young SP; Bali D; DeArmey SM; Mackey J; Kishnani PS
    Mol Genet Metab; 2008 Dec; 95(4):233-5. PubMed ID: 18930676
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study.
    Pena LDM; Barohn RJ; Byrne BJ; Desnuelle C; Goker-Alpan O; Ladha S; Laforêt P; Mengel KE; Pestronk A; Pouget J; Schoser B; Straub V; Trivedi J; Van Damme P; Vissing J; Young P; Kacena K; Shafi R; Thurberg BL; Culm-Merdek K; van der Ploeg AT;
    Neuromuscul Disord; 2019 Mar; 29(3):167-186. PubMed ID: 30770310
    [TBL] [Abstract][Full Text] [Related]  

  • 14. 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. PubMed ID: 25617983
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial.
    Diaz-Manera J; Kishnani PS; Kushlaf H; Ladha S; Mozaffar T; Straub V; Toscano A; van der Ploeg AT; Berger KI; Clemens PR; Chien YH; Day JW; Illarioshkin S; Roberts M; Attarian S; Borges JL; Bouhour F; Choi YC; Erdem-Ozdamar S; Goker-Alpan O; Kostera-Pruszczyk A; Haack KA; Hug C; Huynh-Ba O; Johnson J; Thibault N; Zhou T; Dimachkie MM; Schoser B;
    Lancet Neurol; 2021 Dec; 20(12):1012-1026. PubMed ID: 34800399
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Long-term enzyme-replacement therapy (ERT) with alglucosidase alfa: Evolution of two siblings with juvenile late-onset Pompe disease.
    Rafael Bretón Martínez J; Martínez AC
    J Neurol Sci; 2015 Nov; 358(1-2):459-60. PubMed ID: 26279333
    [No Abstract]   [Full Text] [Related]  

  • 17. Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study.
    van der Ploeg A; Carlier PG; Carlier RY; Kissel JT; Schoser B; Wenninger S; Pestronk A; Barohn RJ; Dimachkie MM; Goker-Alpan O; Mozaffar T; Pena LD; Simmons Z; Straub V; Guglieri M; Young P; Boentert M; Baudin PY; Wens S; Shafi R; Bjartmar C; Thurberg BL
    Mol Genet Metab; 2016 Sep; 119(1-2):115-23. PubMed ID: 27473031
    [TBL] [Abstract][Full Text] [Related]  

  • 18. 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. PubMed ID: 19649685
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Pompe disease: early diagnosis and early treatment make a difference.
    Chien YH; Hwu WL; Lee NC
    Pediatr Neonatol; 2013 Aug; 54(4):219-27. PubMed ID: 23632029
    [TBL] [Abstract][Full Text] [Related]  

  • 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; 68(2):99-109. PubMed ID: 17151339
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 9.