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324 related items for PubMed ID: 32515844

  • 1. Long-term benefit of enzyme replacement therapy with alglucosidase alfa in adults with Pompe disease: Prospective analysis from the French Pompe Registry.
    Semplicini C, De Antonio M, Taouagh N, Béhin A, Bouhour F, Echaniz-Laguna A, Magot A, Nadaj-Pakleza A, Orlikowski D, Sacconi S, Salort-Campana E, Solé G, Tard C, Zagnoli F, Hogrel JY, Hamroun D, Laforêt P, French Pompe Study Group.
    J Inherit Metab Dis; 2020 Nov; 43(6):1219-1231. PubMed ID: 32515844
    [Abstract] [Full Text] [Related]

  • 2. 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; 271(8):5433-5446. PubMed ID: 38896264
    [Abstract] [Full Text] [Related]

  • 3. 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; 31(7):e16292. PubMed ID: 38587143
    [Abstract] [Full Text] [Related]

  • 4. 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; 264(4):621-630. PubMed ID: 27372449
    [Abstract] [Full Text] [Related]

  • 5. Large variation in effects during 10 years of enzyme therapy in adults with Pompe disease.
    Harlaar L, Hogrel JY, Perniconi B, Kruijshaar ME, Rizopoulos D, Taouagh N, Canal A, Brusse E, van Doorn PA, van der Ploeg AT, Laforêt P, van der Beek NAME.
    Neurology; 2019 Nov 05; 93(19):e1756-e1767. PubMed ID: 31619483
    [Abstract] [Full Text] [Related]

  • 6. Respiratory function during enzyme replacement therapy in late-onset Pompe disease: longitudinal course, prognostic factors, and the impact of time from diagnosis to treatment start.
    Stockton DW, Kishnani P, van der Ploeg A, Llerena J, Boentert M, Roberts M, Byrne BJ, Araujo R, Maruti SS, Thibault N, Verhulst K, Berger KI.
    J Neurol; 2020 Oct 05; 267(10):3038-3053. PubMed ID: 32524257
    [Abstract] [Full Text] [Related]

  • 7. Characteristics of Patients With Late-Onset Pompe Disease in France: Insights From the French Pompe Registry in 2022.
    Lefeuvre C, De Antonio M, Bouhour F, Tard C, Salort-Campana E, Lagrange E, Behin A, Sole G, Noury JB, Sacconi S, Magot A, Nadaj-Pakleza A, Lacour A, Beltran S, Spinazzi M, Cintas P, Renard D, Michaud M, Bedat-Millet AL, Prigent H, Taouagh N, Arrassi A, Hamroun D, Attarian S, Laforêt P, for Pompe Study Group.
    Neurology; 2023 Aug 29; 101(9):e966-e977. PubMed ID: 37419682
    [Abstract] [Full Text] [Related]

  • 8. Effect of enzyme replacement therapy with alglucosidase alfa (Myozyme®) in 12 patients with advanced late-onset Pompe disease.
    Papadopoulos C, Orlikowski D, Prigent H, Lacour A, Tard C, Furby A, Praline J, Solé G, Hogrel JY, De Antonio M, Semplicini C, Deibener-Kaminsky J, Kaminsky P, Eymard B, Taouagh N, Perniconi B, Hamroun D, Laforêt P, French Pompe Study Group.
    Mol Genet Metab; 2017 Sep 29; 122(1-2):80-85. PubMed ID: 28648663
    [Abstract] [Full Text] [Related]

  • 9. 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 29; 119(1-2):115-23. PubMed ID: 27473031
    [Abstract] [Full Text] [Related]

  • 10. 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 29; 35(5):837-45. PubMed ID: 22290025
    [Abstract] [Full Text] [Related]

  • 11. 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 29; 25(4):321-32. PubMed ID: 25617983
    [Abstract] [Full Text] [Related]

  • 12. 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]

  • 13. 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 05; 20(12):1012-1026. PubMed ID: 34800399
    [Abstract] [Full Text] [Related]

  • 14. 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 05; 268(7):2482-2492. PubMed ID: 33543425
    [Abstract] [Full Text] [Related]

  • 15. Effect of alglucosidase alfa dosage on survival and walking ability in patients with classic infantile Pompe disease: a multicentre observational cohort study from the European Pompe Consortium.
    Ditters IAM, Huidekoper HH, Kruijshaar ME, Rizopoulos D, Hahn A, Mongini TE, Labarthe F, Tardieu M, Chabrol B, Brassier A, Parini R, Parenti G, van der Beek NAME, van der Ploeg AT, van den Hout JMP, European Pompe Consortium project group on classic infantile Pompe disease.
    Lancet Child Adolesc Health; 2022 Jan 05; 6(1):28-37. PubMed ID: 34822769
    [Abstract] [Full Text] [Related]

  • 16. 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]

  • 17. 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 05; 20(12):1027-1037. PubMed ID: 34800400
    [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 05; 257(1):91-7. PubMed ID: 19649685
    [Abstract] [Full Text] [Related]

  • 19. Cardiopulmonary exercise test to quantify enzyme replacement response in pediatric Pompe disease.
    Bar-Yoseph R, Mandel H, Mainzer G, Gur M, Tal G, Shalloufeh G, Bentur L.
    Pediatr Pulmonol; 2018 Mar 05; 53(3):366-373. PubMed ID: 29356433
    [Abstract] [Full Text] [Related]

  • 20. 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 05; 101(4):338-45. PubMed ID: 20826098
    [Abstract] [Full Text] [Related]

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