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Journal Abstract Search

579 related items for PubMed ID: 28838325

  • 21. Are Anti-rhGAA Antibodies a Determinant of Treatment Outcome in Adults with Late-Onset Pompe Disease? A Systematic Review.
    Ditters IAM, van Kooten HA, van der Beek NAME, van der Ploeg AT, Huidekoper HH, van den Hout JMP.
    Biomolecules; 2023 Sep 19; 13(9):. PubMed ID: 37759814
    [Abstract] [Full Text] [Related]

  • 22. 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 Sep 19; 15():1336599. PubMed ID: 38715621
    [Abstract] [Full Text] [Related]

  • 23. Efficient therapy for refractory Pompe disease by mannose 6-phosphate analogue grafting on acid α-glucosidase.
    Basile I, Da Silva A, El Cheikh K, Godefroy A, Daurat M, Harmois A, Perez M, Caillaud C, Charbonné HV, Pau B, Gary-Bobo M, Morère A, Garcia M, Maynadier M.
    J Control Release; 2018 Jan 10; 269():15-23. PubMed ID: 29108866
    [Abstract] [Full Text] [Related]

  • 24. Antibodies against recombinant human alpha-glucosidase do not seem to affect clinical outcome in childhood onset Pompe disease.
    van Kooten HA, Ditters IAM, Hoogeveen-Westerveld M, Jacobs EH, van den Hout JMP, van Doorn PA, Pijnappel WWMP, van der Ploeg AT, van der Beek NAME.
    Orphanet J Rare Dis; 2022 Feb 02; 17(1):31. PubMed ID: 35109913
    [Abstract] [Full Text] [Related]

  • 25. Improved efficacy of a next-generation ERT in murine Pompe disease.
    Xu S, Lun Y, Frascella M, Garcia A, Soska R, Nair A, Ponery AS, Schilling A, Feng J, Tuske S, Valle MCD, Martina JA, Ralston E, Gotschall R, Valenzano KJ, Puertollano R, Do HV, Raben N, Khanna R.
    JCI Insight; 2019 Mar 07; 4(5):. PubMed ID: 30843882
    [Abstract] [Full Text] [Related]

  • 26. Changes in forced vital capacity over ≤ 13 years among patients with late-onset Pompe disease treated with alglucosidase alfa: new modeling of real-world data from the Pompe Registry.
    Berger KI, Chien YH, Dubrovsky A, Kishnani PS, Llerena JC, Neilan E, Roberts M, Sheng B, Batista JL, Periquet M, Wilson KM, van der Ploeg AT.
    J Neurol; 2024 Aug 07; 271(8):5433-5446. PubMed ID: 38896264
    [Abstract] [Full Text] [Related]

  • 27. The emerging phenotype of late-onset Pompe disease: A systematic literature review.
    Chan J, Desai AK, Kazi ZB, Corey K, Austin S, Hobson-Webb LD, Case LE, Jones HN, Kishnani PS.
    Mol Genet Metab; 2017 Mar 07; 120(3):163-172. PubMed ID: 28185884
    [Abstract] [Full Text] [Related]

  • 28. 104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07).
    Schoser B, Kishnani PS, Bratkovic D, Byrne BJ, Claeys KG, Díaz-Manera J, Laforêt P, Roberts M, Toscano A, van der Ploeg AT, Castelli J, Goldman M, Holdbrook F, Sitaraman Das S, Wasfi Y, Mozaffar T, ATB200-07 Study Group.
    J Neurol; 2024 May 07; 271(5):2810-2823. PubMed ID: 38418563
    [Abstract] [Full Text] [Related]

  • 29. Transcriptomic characterization of clinical skeletal muscle biopsy from late-onset Pompe patients.
    Kinton S, Dufault MR, Zhang M, George K.
    Mol Genet Metab; 2023 Mar 07; 138(3):107526. PubMed ID: 36774918
    [Abstract] [Full Text] [Related]

  • 30. Gene Therapy for Pompe Disease: The Time is now.
    Colella P, Mingozzi F.
    Hum Gene Ther; 2019 Oct 07; 30(10):1245-1262. PubMed ID: 31298581
    [Abstract] [Full Text] [Related]

  • 31. Avalglucosidase alfa: First Approval.
    Dhillon S.
    Drugs; 2021 Oct 07; 81(15):1803-1809. PubMed ID: 34591286
    [Abstract] [Full Text] [Related]

  • 32. Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial.
    Kishnani PS, Diaz-Manera J, Toscano A, Clemens PR, Ladha S, Berger KI, Kushlaf H, Straub V, Carvalho G, Mozaffar T, Roberts M, Attarian S, Chien YH, Choi YC, Day JW, Erdem-Ozdamar S, Illarioshkin S, Goker-Alpan O, Kostera-Pruszczyk A, van der Ploeg AT, An Haack K, Huynh-Ba O, Tammireddy S, Thibault N, Zhou T, Dimachkie MM, Schoser B, COMET Investigator Group.
    JAMA Neurol; 2023 Jun 01; 80(6):558-567. PubMed ID: 37036722
    [Abstract] [Full Text] [Related]

  • 33. 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 01; 22(5):898-907. PubMed ID: 31904026
    [Abstract] [Full Text] [Related]

  • 34. Synergistic Efficacy from Gene Therapy with Coreceptor Blockade and a β2-Agonist in Murine Pompe Disease.
    Han SO, Li S, Bird A, Koeberl D.
    Hum Gene Ther; 2015 Nov 01; 26(11):743-50. PubMed ID: 26417913
    [Abstract] [Full Text] [Related]

  • 35. Increasing Enzyme Mannose-6-Phosphate Levels but Not Miglustat Coadministration Enhances the Efficacy of Enzyme Replacement Therapy in Pompe Mice.
    Anding A, Kinton S, Baranowski K, Brezzani A, De Busser H, Dufault MR, Finn P, Keefe K, Tetrault T, Li Y, Qiu W, Raes K, Vitse O, Zhang M, Ziegler R, Sardi SP, Hunter B, George K.
    J Pharmacol Exp Ther; 2023 Nov 01; 387(2):188-203. PubMed ID: 37679046
    [Abstract] [Full Text] [Related]

  • 36. Duvoglustat HCl Increases Systemic and Tissue Exposure of Active Acid α-Glucosidase in Pompe Patients Co-administered with Alglucosidase α.
    Kishnani P, Tarnopolsky M, Roberts M, Sivakumar K, Dasouki M, Dimachkie MM, Finanger E, Goker-Alpan O, Guter KA, Mozaffar T, Pervaiz MA, Laforet P, Levine T, Adera M, Lazauskas R, Sitaraman S, Khanna R, Benjamin E, Feng J, Flanagan JJ, Barth J, Barlow C, Lockhart DJ, Valenzano KJ, Boudes P, Johnson FK, Byrne B.
    Mol Ther; 2017 May 03; 25(5):1199-1208. PubMed ID: 28341561
    [Abstract] [Full Text] [Related]

  • 37. Long-term benefit of enzyme replacement therapy in Pompe disease: A 5-year prospective study.
    Kuperus E, Kruijshaar ME, Wens SCA, de Vries JM, Favejee MM, van der Meijden JC, Rizopoulos D, Brusse E, van Doorn PA, van der Ploeg AT, van der Beek NAME.
    Neurology; 2017 Dec 05; 89(23):2365-2373. PubMed ID: 29117951
    [Abstract] [Full Text] [Related]

  • 38. Cessation and resuming of alglucosidase alfa in Pompe disease: a retrospective analysis.
    Hundsberger T, Rösler KM, Findling O.
    J Neurol; 2014 Sep 05; 261(9):1684-90. PubMed ID: 24923245
    [Abstract] [Full Text] [Related]

  • 39. Study of the effect of anti-rhGAA antibodies at low and intermediate titers in late onset Pompe patients treated with ERT.
    Fernández-Simón E, Carrasco-Rozas A, Gallardo E, González-Quereda L, Alonso-Pérez J, Belmonte I, Pedrosa-Hernández I, Montiel E, Segovia S, Suárez-Calvet X, Llauger J, Mayos M, Illa I, Barba-Romero MA, Barcena J, Paradas C, Carzorla MR, Creus C, Coll-Cantí J, Díaz M, Domínguez C, Fernández-Torrón R, García-Antelo MJ, Grau JM, López de Munáin A, Martínez-García FA, Morgado Y, Moreno A, Morís G, Muñoz-Blanco MA, Nascimento A, Parajuá-Pozo JL, Querol L, Rojas R, Robledo-Strauss A, Rojas-Marcos Í, Salazar JA, Usón M, Díaz-Manera J.
    Mol Genet Metab; 2019 Sep 05; 128(1-2):129-136. PubMed ID: 31378569
    [Abstract] [Full Text] [Related]

  • 40. 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 05; 149(1):89-97. PubMed ID: 16860134
    [Abstract] [Full Text] [Related]

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