BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

278 related articles for article (PubMed ID: 24923245)

  • 1. 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]  

  • 2. 36-Months follow-up assessment after cessation and resuming of enzyme replacement therapy in late onset Pompe disease: data from the Swiss Pompe Registry.
    Scheidegger O; Leupold D; Sauter R; Findling O; Rösler KM; Hundsberger T
    J Neurol; 2018 Dec; 265(12):2783-2788. PubMed ID: 30232608
    [TBL] [Abstract][Full Text] [Related]  

  • 3. 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]  

  • 4. 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]  

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

  • 6. 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]  

  • 7. 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]  

  • 8. 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
    [TBL] [Abstract][Full Text] [Related]  

  • 9. 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]  

  • 10. 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; 257(1):91-7. PubMed ID: 19649685
    [TBL] [Abstract][Full Text] [Related]  

  • 11. 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]  

  • 12. 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;
    Lancet Neurol; 2021 Dec; 20(12):1012-1026. PubMed ID: 34800399
    [TBL] [Abstract][Full Text] [Related]  

  • 13. 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
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study.
    de Vries JM; van der Beek NA; Hop WC; Karstens FP; Wokke JH; de Visser M; van Engelen BG; Kuks JB; van der Kooi AJ; Notermans NC; Faber CG; Verschuuren JJ; Kruijshaar ME; Reuser AJ; van Doorn PA; van der Ploeg AT
    Orphanet J Rare Dis; 2012 Sep; 7():73. PubMed ID: 23013746
    [TBL] [Abstract][Full Text] [Related]  

  • 15. 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
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Long-term effects of enzyme replacement therapy in an elderly cohort of late-onset Pompe disease.
    Winkler M; von Landenberg C; Kuchenbecker K; Reimann J; Kornblum C
    Neuromuscul Disord; 2022 Mar; 32(3):195-205. PubMed ID: 35120758
    [TBL] [Abstract][Full Text] [Related]  

  • 17. 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
    [TBL] [Abstract][Full Text] [Related]  

  • 18. 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]  

  • 19. Pompe disease in Austria: clinical, genetic and epidemiological aspects.
    Löscher WN; Huemer M; Stulnig TM; Simschitz P; Iglseder S; Eggers C; Moser H; Möslinger D; Freilinger M; Lagler F; Grinzinger S; Reichhardt M; Bittner RE; Schmidt WM; Lex U; Brunner-Krainz M; Quasthoff S; Wanschitz JV
    J Neurol; 2018 Jan; 265(1):159-164. PubMed ID: 29181627
    [TBL] [Abstract][Full Text] [Related]  

  • 20. 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; 268(7):2482-2492. PubMed ID: 33543425
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 14.