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Journal Abstract Search

389 related items for PubMed ID: 28648663

  • 1. 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; 122(1-2):80-85. PubMed ID: 28648663
    [Abstract] [Full Text] [Related]

  • 2. 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
    [Abstract] [Full Text] [Related]

  • 3. 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]

  • 4. 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
    [Abstract] [Full Text] [Related]

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

  • 6. 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]

  • 7. 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]

  • 8. 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]

  • 9. 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]

  • 10. 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]

  • 11. 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]

  • 12. 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 05; 35(5):837-45. PubMed ID: 22290025
    [Abstract] [Full Text] [Related]

  • 13. 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]

  • 14. 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]

  • 15. Observational clinical study in juvenile-adult glycogenosis type 2 patients undergoing enzyme replacement therapy for up to 4 years.
    Angelini C, Semplicini C, Ravaglia S, Bembi B, Servidei S, Pegoraro E, Moggio M, Filosto M, Sette E, Crescimanno G, Tonin P, Parini R, Morandi L, Marrosu G, Greco G, Musumeci O, Di Iorio G, Siciliano G, Donati MA, Carubbi F, Ermani M, Mongini T, Toscano A, Italian GSDII Group.
    J Neurol; 2012 May 20; 259(5):952-8. PubMed ID: 22081099
    [Abstract] [Full Text] [Related]

  • 16. 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]

  • 17. 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 15; 358(1-2):459-60. PubMed ID: 26279333
    [No Abstract] [Full Text] [Related]

  • 18. Observational clinical study of 22 adult-onset Pompe disease patients undergoing enzyme replacement therapy over 5years.
    Stepien KM, Hendriksz CJ, Roberts M, Sharma R.
    Mol Genet Metab; 2016 Apr 15; 117(4):413-8. PubMed ID: 26873529
    [Abstract] [Full Text] [Related]

  • 19. Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease.
    Kanters TA, van der Ploeg AT, Kruijshaar ME, Rizopoulos D, Redekop WK, Rutten-van Mӧlken MPMH, Hakkaart-van Roijen L.
    Orphanet J Rare Dis; 2017 Dec 13; 12(1):179. PubMed ID: 29237491
    [Abstract] [Full Text] [Related]

  • 20. 24-months results in two adults with Pompe disease on enzyme replacement therapy.
    Vielhaber S, Brejova A, Debska-Vielhaber G, Kaufmann J, Feistner H, Schoenfeld MA, Awiszus F.
    Clin Neurol Neurosurg; 2011 Jun 13; 113(5):350-7. PubMed ID: 21477922
    [Abstract] [Full Text] [Related]

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