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.


BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

233 related articles for article (PubMed ID: 21631931)

  • 1. Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy.
    Güngör D; de Vries JM; Hop WC; Reuser AJ; van Doorn PA; van der Ploeg AT; Hagemans ML
    Orphanet J Rare Dis; 2011 Jun; 6():34. PubMed ID: 21631931
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Enzyme replacement therapy reduces the risk for wheelchair dependency in adult Pompe patients.
    van der Meijden JC; Kruijshaar ME; Rizopoulos D; van Doorn PA; van der Beek NAME; van der Ploeg AT
    Orphanet J Rare Dis; 2018 May; 13(1):82. PubMed ID: 29788986
    [TBL] [Abstract][Full Text] [Related]  

  • 3. 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; 12(1):179. PubMed ID: 29237491
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational study.
    Güngör D; Kruijshaar ME; Plug I; D'Agostino RB; Hagemans ML; van Doorn PA; Reuser AJ; van der Ploeg AT
    Orphanet J Rare Dis; 2013 Mar; 8():49. PubMed ID: 23531252
    [TBL] [Abstract][Full Text] [Related]  

  • 5. 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;
    Lancet Child Adolesc Health; 2022 Jan; 6(1):28-37. PubMed ID: 34822769
    [TBL] [Abstract][Full Text] [Related]  

  • 6. 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
    [TBL] [Abstract][Full Text] [Related]  

  • 7. 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; 89(23):2365-2373. PubMed ID: 29117951
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Pompe disease: early diagnosis and early treatment make a difference.
    Chien YH; Hwu WL; Lee NC
    Pediatr Neonatol; 2013 Aug; 54(4):219-27. PubMed ID: 23632029
    [TBL] [Abstract][Full Text] [Related]  

  • 9. [Pompe disease treated with enzyme replacement therapy in pregnancy].
    Grosz Z; Várdi KV; Molnár JM
    Ideggyogy Sz; 2020 Sep; 73(9-10):339-344. PubMed ID: 33035415
    [TBL] [Abstract][Full Text] [Related]  

  • 10. 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; 267(10):3038-3053. PubMed ID: 32524257
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Enzyme replacement therapy and fatigue in adults with Pompe disease.
    Güngör D; de Vries JM; Brusse E; Kruijshaar ME; Hop WC; Murawska M; van den Berg LE; Reuser AJ; van Doorn PA; Hagemans ML; Plug I; van der Ploeg AT
    Mol Genet Metab; 2013 Jun; 109(2):174-8. PubMed ID: 23603069
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Ten years of the international Pompe survey: patient reported outcomes as a reliable tool for studying treated and untreated children and adults with non-classic Pompe disease.
    van der Meijden JC; Güngör D; Kruijshaar ME; Muir AD; Broekgaarden HA; van der Ploeg AT
    J Inherit Metab Dis; 2015 May; 38(3):495-503. PubMed ID: 25112389
    [TBL] [Abstract][Full Text] [Related]  

  • 13. 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;
    Neurology; 2023 Aug; 101(9):e966-e977. PubMed ID: 37419682
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Quality of life and participation in daily life of adults with Pompe disease receiving enzyme replacement therapy: 10 years of international follow-up.
    Güngör D; Kruijshaar ME; Plug I; Rizopoulos D; Kanters TA; Wens SC; Reuser AJ; van Doorn PA; van der Ploeg AT
    J Inherit Metab Dis; 2016 Mar; 39(2):253-60. PubMed ID: 26531313
    [TBL] [Abstract][Full Text] [Related]  

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

  • 16. 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;
    J Inherit Metab Dis; 2020 Nov; 43(6):1219-1231. PubMed ID: 32515844
    [TBL] [Abstract][Full Text] [Related]  

  • 17. 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;
    Mol Genet Metab; 2017 Sep; 122(1-2):80-85. PubMed ID: 28648663
    [TBL] [Abstract][Full Text] [Related]  

  • 18. 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; 93(19):e1756-e1767. PubMed ID: 31619483
    [TBL] [Abstract][Full Text] [Related]  

  • 19. 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;
    J Neurol; 2012 May; 259(5):952-8. PubMed ID: 22081099
    [TBL] [Abstract][Full Text] [Related]  

  • 20. 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; 35(5):837-45. PubMed ID: 22290025
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 12.