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Journal Abstract Search

389 related items for PubMed ID: 28648663

  • 21. Effects of short-to-long term enzyme replacement therapy (ERT) on skeletal muscle tissue in late onset Pompe disease (LOPD).
    Ripolone M, Violano R, Ronchi D, Mondello S, Nascimbeni A, Colombo I, Fagiolari G, Bordoni A, Fortunato F, Lucchini V, Saredi S, Filosto M, Musumeci O, Tonin P, Mongini T, Previtali S, Morandi L, Angelini C, Mora M, Sandri M, Sciacco M, Toscano A, Comi GP, Moggio M.
    Neuropathol Appl Neurobiol; 2018 Aug; 44(5):449-462. PubMed ID: 28574618
    [Abstract] [Full Text] [Related]

  • 22. 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
    [Abstract] [Full Text] [Related]

  • 23. Long term clinical history of an Italian cohort of infantile onset Pompe disease treated with enzyme replacement therapy.
    Parini R, De Lorenzo P, Dardis A, Burlina A, Cassio A, Cavarzere P, Concolino D, Della Casa R, Deodato F, Donati MA, Fiumara A, Gasperini S, Menni F, Pagliardini V, Sacchini M, Spada M, Taurisano R, Valsecchi MG, Di Rocco M, Bembi B.
    Orphanet J Rare Dis; 2018 Feb 08; 13(1):32. PubMed ID: 29422078
    [Abstract] [Full Text] [Related]

  • 24.
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  • 25. Motor and respiratory decline in patients with late onset Pompe disease after cessation of enzyme replacement therapy during COVID-19 pandemic.
    Tard C, Salort-Campana E, Michaud M, Spinazzi M, Nadaj Pakleza A, Durr H, Bouhour F, Lefeuvre C, Thomas R, Arrassi A, Taouagh N, Pompe Study Group, Solé G, Laforêt P.
    Eur J Neurol; 2022 Apr 08; 29(4):1181-1186. PubMed ID: 34927321
    [Abstract] [Full Text] [Related]

  • 26. Survival and long-term outcomes in late-onset Pompe disease following alglucosidase alfa treatment: a systematic review and meta-analysis.
    Schoser B, Stewart A, Kanters S, Hamed A, Jansen J, Chan K, Karamouzian M, Toscano A.
    J Neurol; 2017 Apr 08; 264(4):621-630. PubMed ID: 27372449
    [Abstract] [Full Text] [Related]

  • 27. 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 Apr 08; 104(1-2):118-22. PubMed ID: 21802969
    [Abstract] [Full Text] [Related]

  • 28. White matter lesions in treated late onset Pompe disease are not different to matched controls.
    Schneider I, Hensel O, Zierz S.
    Mol Genet Metab; 2019 Jun 08; 127(2):128-131. PubMed ID: 31153821
    [Abstract] [Full Text] [Related]

  • 29. 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 08; 271(8):5433-5446. PubMed ID: 38896264
    [Abstract] [Full Text] [Related]

  • 30. Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients.
    Masat E, Laforêt P, De Antonio M, Corre G, Perniconi B, Taouagh N, Mariampillai K, Amelin D, Mauhin W, Hogrel JY, Caillaud C, Ronzitti G, Puzzo F, Kuranda K, Colella P, Mallone R, Benveniste O, Mingozzi F, French Pompe Registry Study Group.
    Sci Rep; 2016 Nov 04; 6():36182. PubMed ID: 27812025
    [Abstract] [Full Text] [Related]

  • 31. 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 04; 139(3):107611. PubMed ID: 37285781
    [Abstract] [Full Text] [Related]

  • 32. 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 04; 20(12):1012-1026. PubMed ID: 34800399
    [Abstract] [Full Text] [Related]

  • 33. Early higher dosage of alglucosidase alpha in classic Pompe disease.
    Spada M, Pagliardini V, Ricci F, Biamino E, Mongini T, Porta F.
    J Pediatr Endocrinol Metab; 2018 Dec 19; 31(12):1343-1347. PubMed ID: 30433875
    [Abstract] [Full Text] [Related]

  • 34. New therapeutic approaches for Pompe disease: enzyme replacement therapy and beyond.
    Kishnani PS, Beckemeyer AA.
    Pediatr Endocrinol Rev; 2014 Sep 19; 12 Suppl 1():114-24. PubMed ID: 25345093
    [Abstract] [Full Text] [Related]

  • 35. Alglucosidase alfa: new drug. Pompe disease: a short-term benefit.
    Prescrire Int; 2007 Dec 19; 16(92):240-1. PubMed ID: 18092404
    [Abstract] [Full Text] [Related]

  • 36. Assessing the Role of Anti rh-GAA in Modulating Response to ERT in a Late-Onset Pompe Disease Cohort from the Italian GSDII Study Group.
    Filosto M, Cotti Piccinelli S, Ravaglia S, Servidei S, Moggio M, Musumeci O, Donati MA, Pegoraro E, Di Muzio A, Maggi L, Tonin P, Marrosu G, Sancricca C, Lerario A, Sacchini M, Semplicini C, Bozzoni V, Telese R, Bonanno S, Piras R, Maioli MA, Ricci G, Vercelli L, Galvagni A, Gallo Cassarino S, Caria F, Mongini T, Siciliano G, Padovani A, Toscano A.
    Adv Ther; 2019 May 19; 36(5):1177-1189. PubMed ID: 30879255
    [Abstract] [Full Text] [Related]

  • 37. Aerobic training as an adjunctive therapy to enzyme replacement in Pompe disease.
    Nilsson MI, Samjoo IA, Hettinga BP, Koeberl DD, Zhang H, Hawke TJ, Nissar AA, Ali T, Brandt L, Ansari MU, Hazari H, Patel N, Amon J, Tarnopolsky MA.
    Mol Genet Metab; 2012 Nov 19; 107(3):469-79. PubMed ID: 23041258
    [Abstract] [Full Text] [Related]

  • 38. STIG study: real-world data of long-term outcomes of adults with Pompe disease under enzyme replacement therapy with alglucosidase alfa.
    Gutschmidt K, Musumeci O, Díaz-Manera J, Chien YH, Knop KC, Wenninger S, Montagnese F, Pugliese A, Tavilla G, Alonso-Pérez J, Hwu PW, Toscano A, Schoser B.
    J Neurol; 2021 Jul 19; 268(7):2482-2492. PubMed ID: 33543425
    [Abstract] [Full Text] [Related]

  • 39. Enzyme replacement therapy with alglucosidase alfa in a late-onset Pompe disease patient during pregnancy.
    Oliveira Santos M, Evangelista T, Conceição I.
    Neuromuscul Disord; 2018 Nov 19; 28(11):965-968. PubMed ID: 30314719
    [Abstract] [Full Text] [Related]

  • 40. Real-life effectiveness 1 year after switching to avalglucosidase alfa in late-onset Pompe disease patients worsening on alglucosidase alfa therapy: A French cohort study.
    Tard C, Bouhour F, Michaud M, Beltran S, Fournier M, Demurger F, Lagrange E, Nollet S, Sacconi S, Noury JB, Magot A, Cintas P, Renard D, Deibener-Kaminsky J, Lefeuvre C, Davion JB, Salort-Campana E, Arrassi A, Taouagh N, Spinazzi M, Pompe Study Group, Attarian S, Laforêt P.
    Eur J Neurol; 2024 Jul 19; 31(7):e16292. PubMed ID: 38587143
    [Abstract] [Full Text] [Related]

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