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.


Pubmed for Handhelds

PUBMED FOR HANDHELDS

Journal Abstract Search

327 related items for PubMed ID: 29565424

  • 1. 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, Pompe ADVANCE Study Consortium.
    Genet Med; 2018 Oct; 20(10):1284-1294. PubMed ID: 29565424
    [Abstract] [Full Text] [Related]

  • 2. Clinical characteristics and genotypes in the ADVANCE baseline data set, a comprehensive cohort of US children and adolescents with Pompe disease.
    Kishnani PS, Gibson JB, Gambello MJ, Hillman R, Stockton DW, Kronn D, Leslie ND, Pena LDM, Tanpaiboon P, Day JW, Wang RY, Goldstein JL, An Haack K, Sparks SE, Zhao Y, Hahn SH, Pompe ADVANCE Study Consortium.
    Genet Med; 2019 Nov; 21(11):2543-2551. PubMed ID: 31086307
    [Abstract] [Full Text] [Related]

  • 3. Optimizing treatment outcomes: immune tolerance induction in Pompe disease patients undergoing enzyme replacement therapy.
    Chen HA, Hsu RH, Fang CY, Desai AK, Lee NC, Hwu WL, Tsai FJ, Kishnani PS, Chien YH.
    Front Immunol; 2024 Nov; 15():1336599. PubMed ID: 38715621
    [Abstract] [Full Text] [Related]

  • 4. 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, COMET Investigator Group.
    Lancet Neurol; 2021 Dec; 20(12):1012-1026. PubMed ID: 34800399
    [Abstract] [Full Text] [Related]

  • 5. Enzyme replacement therapy for infantile-onset Pompe disease.
    Chen M, Zhang L, Quan S.
    Cochrane Database Syst Rev; 2017 Nov 20; 11(11):CD011539. PubMed ID: 29155436
    [Abstract] [Full Text] [Related]

  • 6. An immune tolerance approach using transient low-dose methotrexate in the ERT-naïve setting of patients treated with a therapeutic protein: experience in infantile-onset Pompe disease.
    Kazi ZB, Desai AK, Troxler RB, Kronn D, Packman S, Sabbadini M, Rizzo WB, Scherer K, Abdul-Rahman O, Tanpaiboon P, Nampoothiri S, Gupta N, Feigenbaum A, Niyazov DM, Sherry L, Segel R, McVie-Wylie A, Sung C, Joseph AM, Richards S, Kishnani PS.
    Genet Med; 2019 Apr 20; 21(4):887-895. PubMed ID: 30214072
    [Abstract] [Full Text] [Related]

  • 7. A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe disease.
    Byrne BJ, Geberhiwot T, Barshop BA, Barohn R, Hughes D, Bratkovic D, Desnuelle C, Laforet P, Mengel E, Roberts M, Haroldsen P, Reilley K, Jayaram K, Yang K, Walsh L, POM-001/002 Investigators.
    Orphanet J Rare Dis; 2017 Aug 24; 12(1):144. PubMed ID: 28838325
    [Abstract] [Full Text] [Related]

  • 8. 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, NEO1 Investigator Group.
    Neuromuscul Disord; 2019 Mar 24; 29(3):167-186. PubMed ID: 30770310
    [Abstract] [Full Text] [Related]

  • 9. Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease.
    Dimachkie MM, Barohn RJ, Byrne B, Goker-Alpan O, Kishnani PS, Ladha S, Laforêt P, Mengel KE, Peña LDM, Sacconi S, Straub V, Trivedi J, Van Damme P, van der Ploeg AT, Vissing J, Young P, Haack KA, Foster M, Gilbert JM, Miossec P, Vitse O, Zhou T, Schoser B, NEO-EXT investigators.
    Neurology; 2022 Aug 01; 99(5):e536-e548. PubMed ID: 35618441
    [Abstract] [Full Text] [Related]

  • 10. 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 01; 11(3):210-9. PubMed ID: 19287243
    [Abstract] [Full Text] [Related]

  • 11. 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 01; 149(1):89-97. PubMed ID: 16860134
    [Abstract] [Full Text] [Related]

  • 12. Cardiac responses in paediatric Pompe disease in the ADVANCE patient cohort.
    Byrne BJ, Colan SD, Kishnani PS, Foster MC, Sparks SE, Gibson JB, An Haack K, Stockton DW, Peña LDM, Hahn SH, Johnson J, Tanpaiboon PX, Leslie ND, Kronn D, Hillman RE, Wang RY, Pompe ADVANCE Study Consortium.
    Cardiol Young; 2022 Mar 01; 32(3):364-373. PubMed ID: 34420548
    [Abstract] [Full Text] [Related]

  • 13. 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 01; 107(3):456-61. PubMed ID: 23031366
    [Abstract] [Full Text] [Related]

  • 14. 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 09; 68(2):99-109. PubMed ID: 17151339
    [Abstract] [Full Text] [Related]

  • 15.
    Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Amemiya A, Leslie N, Bailey L.
    ; 1993 Jan 09. PubMed ID: 20301438
    [Abstract] [Full Text] [Related]

  • 16. Long-Term Experience with Anaphylaxis and Desensitization to Alglucosidase Alfa in Pompe Disease.
    Ertoy Karagol HI, Inci A, Terece SP, Kilic A, Demir F, Yapar D, Koken G, Okur I, Ezgu FS, Tumer L, Bakirtas A, Gazi University Enzyme Replacement Therapy Hypersensitivity Study Group.
    Int Arch Allergy Immunol; 2023 Jan 09; 184(4):370-375. PubMed ID: 36623499
    [Abstract] [Full Text] [Related]

  • 17. Higher dosing of alglucosidase alfa improves outcomes in children with Pompe disease: a clinical study and review of the literature.
    Khan AA, Case LE, Herbert M, DeArmey S, Jones H, Crisp K, Zimmerman K, ElMallah MK, Young SP, Kishnani PS.
    Genet Med; 2020 May 09; 22(5):898-907. PubMed ID: 31904026
    [Abstract] [Full Text] [Related]

  • 18. Immune Tolerance-Adjusted Personalized Immunogenicity Prediction for Pompe Disease.
    De Groot AS, Desai AK, Lelias S, Miah SMS, Terry FE, Khan S, Li C, Yi JS, Ardito M, Martin WD, Kishnani PS.
    Front Immunol; 2021 May 09; 12():636731. PubMed ID: 34220802
    [Abstract] [Full Text] [Related]

  • 19. Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial.
    Schoser B, Roberts M, Byrne BJ, Sitaraman S, Jiang H, Laforêt P, Toscano A, Castelli J, Díaz-Manera J, Goldman M, van der Ploeg AT, Bratkovic D, Kuchipudi S, Mozaffar T, Kishnani PS, PROPEL Study Group.
    Lancet Neurol; 2021 Dec 09; 20(12):1027-1037. PubMed ID: 34800400
    [Abstract] [Full Text] [Related]

  • 20. Higher dose alglucosidase alfa is associated with improved overall survival in infantile-onset Pompe disease (IOPD): data from the Pompe Registry.
    Kishnani PS, Kronn D, Suwazono S, Broomfield A, Llerena J, Al-Hassnan ZN, Batista JL, Wilson KM, Periquet M, Daba N, Hahn A, Chien YH.
    Orphanet J Rare Dis; 2023 Dec 06; 18(1):381. PubMed ID: 38057861
    [Abstract] [Full Text] [Related]

    Page: [Next] [New Search]
    of 17.